jarofhope

Understanding the FDA’s New ELEVIDYS Warning: A Family-Centered Perspective

Tue, 18 Nov 2025 17:58:25 GMT

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Agamree becomes 1st approved DMD treatment in Canada

Thu, 09 Oct 2025 15:56:58 GMT

Health Canada gives OK to therapy for patients 4 and older

Health Canada has approved Agamree (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients ages 4 and older, making Agamree the first approved DMD treatment in Canada.

Canada’s approval is the sixth for Agamree to treat DMD. The therapy is approved in the U.S. , the U.K. , and the European Union , as well as in China and Hong Kong, according to a press release from the therapy’s developer, Santhera Pharmaceuticals .

DMD is caused by mutations in the gene that provides instructions to make dystrophin, a large protein that acts like a shock absorber in muscle cells to cushion cells against damage. People with DMD produce virtually no functional dystrophin protein, and as a result, muscles accumulate excessive wear-and-tear damage over time, leading to MD symptoms like muscle weakness and wasting.

DMD treatment in Canada has relied largely on corticosteroids, anti-inflammatory medications that work by mimicking the stress hormone cortisol. By binding to cortisol receptors on the body’s cells, corticosteroids can help reduce muscle inflammation to preserve muscle function in DMD. However, long-term corticosteroid use can lead to a host of side effects including weight gain, skin issues, and mood changes.

Treatment designed to work with fewer side effects

The active agent in Agamree is a so-called dissociative corticosteroid. It’s designed to bind to cortisol receptors in a way that recapitulates the anti-inflammatory properties of traditional corticosteroids, but with fewer side effects.

Approvals of Agamree have been based largely on data the pivotal Phase 2b VISION-DMD clinical trial (NCT03439670) . The study tested Agamree against prednisone, a commonly used corticosteroid, in DMD patients. Results showed that both treatments were comparable in terms of their ability to preserve muscle function , but Agamree was associated with fewer side effects , reversal of stunted growth, and stabilized body mass index (a measure of body fat that accounts for weight and height).

Agamree was originally developed by Reveragen Biopharma . Catalyst Pharmaceuticals secured commercial rights to Agamree in North America in a 2023 deal with Santhera.

Agamree will be sold in Canada by Kye Pharmaceuticals , which secured Canadian rights to the therapy in a deal with Catalyst .

Kye’s application seeking approval of Agamree was granted priority review by Health Canada, shortening the time it takes to reach a decision to six months from the standard 10 months.

Disability advocates let their voices be heard at symposium

Wed, 08 Oct 2025 15:59:46 GMT

People with Duchenne MD, other diseases have a place in shaping the future

A couple weeks ago, I co-emceed the Zero Project Asia Pacific Symposium 2025 with my girlfriend, Amanda. The event, a collaboration between SG Enable and the Zero Project, took place at Enabling Village, a community space here in Singapore designed for accessibility.

The symposium gathered leaders in Asia to share ideas on how to remove barriers for people with disabilities. For the two of us, it was a chance to stand together as disabled advocates, side by side.

Amanda lives with progressive vision loss, and I live with Duchenne muscular dystrophy (DMD), a genetic condition that weakens muscles. By the age of 30, many of us with Duchenne rely fully on wheelchairs, ventilators , and round-the-clock caregiving . Some do not survive into adulthood. That reality was never far from my mind as I was being wheeled onto the stage with my BiPAP ventilator attached behind me.

Why Duchenne stories need global platforms

Public forums can sometimes feel distant from everyday life , with discussions full of policy and strategy. But for me, being part of it carried a different weight. Sitting in front of a room filled with government officials, nonprofit leaders, and fellow advocates , I thought about how Duchenne shapes every part of my day, from the strength I no longer have to the planning required for something as simple as leaving my home.

When Amanda and I introduced speakers, we carried with us the lived experiences of people who are often unseen. By being visible on that stage, we reminded the audience that policies must serve real lives.

The symposium organizers built the event on the Zero Project ’s work. According to its website, the project, “founded by the Austrian nonprofit Essl Foundation in 2008, … is a global, research-driven initiative to advance the implementation of the United Nations’ Convention on the Rights of Persons with Disabilities by finding and sharing innovative solutions that remove barriers for persons with disabilities. Over the past decade, the Zero Project has established a global network of more than 10,000 experts, policymakers, corporations, academics, and people with and without disabilities. This network contributes to the Zero Project’s work and continues to grow, fostering a global community for disability inclusion.”

Singapore’s Ministry of Social and Family Development set up SG Enable , Singapore’s focal agency for disability inclusion, in 2013. SG Enable works to create fair opportunities for people with disabilities to live, learn, work, and play in an inclusive society. Guided by professionalism, innovation, and empathy, it strives to build a more inclusive Singapore.

Representing the Duchenne community in public conversations

In Singapore, where people still often hide away disabilities, two disabled people emceed the Zero Project Asia Pacific Symposium 2025 , sending a powerful message that voices like ours belong at the center of conversations about inclusion.

I don’t know how long Duchenne will allow me to keep doing this work, But I do know this: Every time I’m given the microphone, I’ll speak not just for myself, but for the global Duchenne community. My brother, Isaac, who died at 28 from Duchenne, never got the chance to stand on a stage like this. I carry him with me whenever I speak.

That day at the symposium, Amanda and I proved that people with Duchenne and other disabilities have a place in shaping the future — not in the background, but right there in the spotlight.

Note: Muscular Dystrophy News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis , or treatment . This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Muscular Dystrophy News Today or its parent company, Bionews, and are intended to spark discussion about issues pertaining to muscular dystrophy .

Exon-skipping therapy shows promise for DMD44

Tue, 07 Oct 2025 16:02:24 GMT

Del-zota helps ambulatory, nonambulatory patients, trial data show

Delpacibart zotadirsen (del-zota), an investigational exon-skipping therapy from Avidity Biosciences , is demonstrating the potential to reverse disease progression in boys and young men with Duchenne muscular dystrophy (DMD) amenable to exon 44 skipping, or DMD44.

That’s according to one-year data from the Phase 1/2 EXPLORE44 trial (NCT05670730) and the EXPLORE44 open-label extension (OLE) study (NCT06244082) , which showed that the treatment led to functional improvements for DMD patients who both could (ambulatory) and could not (nonambulatory) walk.

“These results represent an important step forward for the DMD community and offer hope to people and families affected by DMD44 that del-zota may … result in meaningful, real-world impact,” Steve Hughes, MD, chief medical officer for Avidity, said in a written Q&A with Muscular Dystrophy News Today.

The DMD community is similarly enthusiastic. In a separate statement, Michael Kelly, PhD, chief scientific officer for CureDuchenne , said that the nonprofit, which has invested in the early clinical development of del-zota, “is thrilled to see these results.”

Del-zota’s “approval can’t come soon enough for Duchenne patients awaiting meaningful treatment options,” Kelly said.

On track for accelerated approval application

Avidity is on track to seek accelerated approval of del-zota from the U.S. Food and Drug Administration (FDA) by year’s end. Regulators have granted the therapy several statuses intended to speed its clinical development over the years, and have aligned with Avidity on its plans for accelerated approval.

Hughes said the company is “focused on bringing del-zota to people living with DMD44 as quickly as possible,” while working closely with regulators to ensure top safety and quality standards.

“FDA accelerated approval of del-zota would represent a major step forward for the DMD community, helping to potentially expand treatment options for these patients and paving the way for further innovation,” the scientist said.

While accelerated approval would allow del-zota to be marketed based on preliminary clinical trial data, conversion to full approval will require more evidence of the therapy’s clinical benefit.

To that end, top-line data from the EXPLORE44 OLE study are due later this year. Plans are also underway for a Phase 3 confirmatory trial, which Avidity will share with regulators “in the very near future,” Hughes said.

Trial shows treatment boosts dystrophin production

People with DMD have mutations in the DMD gene that lead to a deficiency in a muscle-protecting protein called dystrophin, driving the hallmark symptoms of muscle weakness and wasting.

The DMD gene includes protein-coding segments called exons. Like other exon-skipping therapies , del-zota works to mask an exon, enabling production of a shorter but still functional dystrophin protein. It is intended for people with mutations amenable to exon 44 skipping, and is given via infusions into the bloodstream.

EXPLORE44 enrolled boys and men with DMD, ages 7-27, who each received three doses of del-zota or a placebo at either 5 mg/kg every six weeks or 10 mg/kg every eight weeks. The OLE involves 39 participants, 23 of whom rolled over from EXPLORE44. All are receiving the 5 mg/kg regimen for two years.

Avidity recently reported results from 17 participants, 12 ambulatory and five nonambulatory, who were treated with del-zota continuously for one year across the EXPLORE44 and EXPLORE44-OLE studies.

Consistent with earlier reports, the data showed that del-zota led to statistically significant increases in dystrophin production by about 25% of normal. Total dystrophin levels were restored to up to 58% of normal.

Levels of creatine kinase, a biomarker of muscle damage, rapidly declined by more than 80% and were sustained at near normal levels with up to 16 months of follow-up. About half of the participants had creatine kinase levels within the normal range after a year.

While the biomarker data is promising, “it was important for us to learn how these results may translate into functional improvements,” Hughes noted.

Across several functional evaluations, including ones assessing both upper and lower limb function, the data showed consistent improvements for patients treated with del-zota compared with a group of untreated participants from a separate natural study, in whom functional abilities declined over time.

These gains were observed for both ambulatory and nonambulatory patients, Hughes noted.

The data overall “are indicative of a reversal in disease progression” relative to what’s expected in the disease’s natural course, Kelly said.

Several exon-skipping therapies are already on the U.S. market for treating DMD, although none is designed for exon 44 skipping.

While it has been “incredible to see how the field has evolved in recent years,” first-generation exon-skipping therapies face notable delivery challenges, Hughes said. When given into the bloodstream, they don’t easily enter muscle cells, where they’re most needed.

Del-zota, known as an antibody oligonucleotide conjugate (AOC), is designed to overcome that challenge. It contains a molecule used to skip exon 44 called a phosphorodiamidate morpholino oligomer (PMO), linked to an antibody that targets the treatment to skeletal and cardiac muscles.

In clinical studies, del-zota has led to better muscle delivery of the PMO than any other similar technology to date, according to Hughes.

The clinical development of some other PMO-based investigational therapies for DMD has been discontinued over the last year. That del-zota has demonstrated a good long-term safety profile is “very reassuring,” Kelly noted. The most common treatment-emergent side effects in the recent analysis were upper respiratory tract symptoms, diarrhea, fall, back pain, and headache, which were usually mild or moderate.

Avidity believes its AOC platform ultimately holds promise for treating a wider range of muscular dystrophy (MD) patients.

It has a number of candidates in development to target additional DMD-causing mutations. One called AOC 1045, intended for exon 45 skipping, is next in line for clinical trials, according to Hughes.

The company is also working on therapies for less common forms of MD for which there are no approved treatments, including delpacibart etedesiran (del-desiran) for myotonic dystrophy type 1 and delpacibart braxlosiran (del-brax) for facioscapulohumeral MD .

A completely enrolled Phase 3 trial of del-desiran called HARBOR (NCT06411288) is underway, and a Phase 3 trial of del-brax called FORTITUDE-3 (NCT07038200) is recruiting at U.S. sites. Avidity intends to seek regulatory approvals for both therapies next year.

“When one therapy succeeds, we all succeed,” Hughes said. “We hope that our momentum … encourages further innovation in developing targeted delivery technologies … potentially leading to more treatments for people in need who are living with rare and serious diseases.”

Latest News with Sareptas Drug for the Duchenne Community

Thu, 31 Jul 2025 17:14:46 GMT

�� What Happened

FDA Request: On July 18, the FDA informally asked Sarepta to voluntarily halt all U.S. shipments of its Duchenne DMD gene therapy, Elevidys—a move driven by safety concerns after three patient deaths due to acute liver failure. This included two fatalities in teenage Duchenne patients treated with Elevidys and a third in an adult patient from a different Sarepta gene therapy trial .

Initial Pushback: Sarepta initially resisted, stating there were no new safety concerns for ambulatory patients (those who can walk), and maintained shipments for that group. They had previously paused shipments for non-ambulatory patients earlier in the year after two deaths .

Reversal and Pause: On July 21–22, Sarepta reversed course and voluntarily paused all shipments in the U.S. by end of business Tuesday, July 22, 2025, to cooperate with the FDA, respond to inquiries, and complete a revised safety labeling for Elevidys .

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�� Why It Matters

Safety Labeling Update: The pause allows Sarepta and the FDA time to add a stricter warning (including a new black-box liver-risk warning) and possibly implement a Risk Evaluation and Mitigation Strategy (REMS) for monitoring liver safety .

Regulatory Relations: CEO Doug Ingram labeled the decision “painful,” but necessary for maintaining a collaborative and constructive relationship with the FDA .

Market Impact: Sarepta’s stock plunged, falling as much as 8–5% on the news, and is down nearly 90% year to date. Analysts warn that the pause and black-box warning could severely affect Elevidys uptake and 50–61% of Sarepta's revenue .

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�� What’s Next

Duration of Pause: The pause could extend 3–6 months while safety data is reviewed and label changes are finalized .

Potential Outcomes:

Resumption: Elevidys could return under new safety protocols/label. A REMS program may be required .

Market Withdrawal: Analysts caution on the possibility of permanent withdrawal if FDA's safety concerns aren't resolved .

Pipeline Impact: FDA has also placed a clinical hold on Sarepta’s other gene therapy trials (including for Limb-Girdle Muscular Dystrophy) and revoked platform tech designation due to the shared AAVrh74 viral vector .

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✅ In Summary

Key Takeaway Details

Pause in effect All U.S. Elevidys shipments halted since end of business July 22

Why Safety concerns after 3 liver-related deaths from same viral vector

Next steps Label update, FDA review, possible REMS; uncertain pause duration

Risks Potential permanent withdrawal, impact on revenue, and pipeline delays

Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys

Fri, 20 Jun 2025 15:29:36 GMT

This article originally appeared on www.statnews.com , written by Christine McSherry—a registered nurse and founder of the Jett Foundation—on June 18, 2025.

On Sunday at 1 a.m. Eastern time, an announcement went out from the first-generation gene therapy company for Duchenne muscular dystrophy: A second patient who had been treated with Elevidys has died .

This second family said yes to hope, yes to science, yes to the risk, and is now grieving the irreversible outcome of an irreversible drug. Others who also said “yes” are living with consequences from adverse events that we still don’t fully understand: cardiac complications, thrombocytopenia, nausea, vomiting, and elevated liver enzymes.

These families — our families — deserve more.

I know firsthand how difficult it is to make decisions about a cutting-edge treatment, to balance the risks and potential rewards. My son Jett, the third of my five children, was diagnosed with Duchenne in 2001 at the age of 5. In 2012, I activated an exploratory qualitative program to better understand meaningful benefits that were being seen in Sarepta’s study of eteplirsen. I found that patients were benefiting from eteplirsen but, as with the disease, in heterogeneous ways that were not being captured by the more clinician-directed traditional assessments. Eteplirsen, now Exondys 51, was approved by the FDA in 2016 .

Nearly 10 years later, the landscape has changed fundamentally. I am overwhelmed by the promise of gene therapy. We need to protect that promise — not by slowing innovation or fast approvals and access to life-changing medications, but by making the system work better. That begins with data, safety, and respect for those who took the biggest risk.

Duchenne muscular dystrophy is a rare muscle-wasting condition that causes muscle weakness, loss of mobility, and early death in mostly boys and young men. There are girls also with Duchenne, though they are much more rare.

Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin — the protein missing in those with Duchenne — into patient cells. The cost of the one-time infusion is $3.2 million.

The sponsor, Sarepta Therapeutics, received accelerated approval in June 2023 from the Food and Drug Administration for the use of Elevidys in ambulatory boys ages 4 and 5 years old. On June 20, 2024, Peter Marks, then the director of the FDA’s Center for Biologics Evaluation and Research, broadened the approval of Elevidys to traditional full approval for boys 4 and older and continued accelerated approval for non-ambulatory patients 4 and older.

In mid-June, Sarepta reported that more than 900 patients have received Elevidys globally, leading to more than $800 million in revenue.

We need to acknowledge just how agonizing these decisions are — and were — made by families in our community. Elevidys gave to the Duchenne community so much hope, but also risk. Decisions to participate in a clinical trial or access the commercial drug are not made on paper. They are made in private moments of fear, love, and hope by caregivers and patients who are living with a life-altering and life-limiting disorder.

But the reality is, patients who are treated — ambulatory and non-ambulatory alike — are at risk for serious complications and, sadly, death.

And while Sarepta brought us hope, it has also silenced concerns, has not fully disclosed adverse events, and appears to be operating in the best interests of its investors, not patients and families. Many of us find that we get our most reliable information from each other, from social media, where people share their experiences with Elevidys.

The FDA created the Adverse Event Reporting System ( FAERS ) dashboard for good reason. According to the FDA website, the purpose is to expand FAERS data access so that the general public can search for information related to human adverse events reported to FDA by the pharmaceutical industry, health care providers, and consumers. It helps us understand the risks associated with medications. The data is the responsibility of several stakeholders, including the sponsor.

However, information is missing from the Elevidys data, including patients’ ages and weight. That’s important because many in the Duchenne community want to understand if these deadly complications exist only in patients who are no longer ambulatory. Knowing a patient’s age can be extremely useful. In addition, during an investor call and update Monday morning, the CEO said that there was no evidence to also halt distribution of Elevidys for ambulatory participants.

But the FAERS dashboard confirms what the Duchenne community is reading on social media pages: Ambulatory boys have significant adverse events, including elevated liver enzymes that require complex, urgent, multidisciplinary medical care. Has the bar now been set so that only death triggers a serious evaluation of the safety of a new, irreversible gene therapy? These patients are undergoing days to weeks of multiple hospitalizations, high doses of IV steroids, rounds of intravenous immunoglobulin treatment, and when all that fails or is simply not enough, months of sirolimus, also known by the brand name Rapamune, a powerful immunosuppressant drug primarily used in renal transplant patients to help prevent their bodies from rejecting the new organ.

Given the pain, uncertainty, and expense of the treatment, we should be able to get information from the many conferences that we attend, the FAERS dashboard, and real-time communication with clearly written updates from the sponsor to help the community make educated choices. The burden of clarity, transparency, and proactive communication should fall on the sponsor — not the families.

But instead, we are stuck with social media. Caregivers of boys treated with Elevidys are sharing a growing volume of anecdotal safety and outcome data. These real-world reports — often posted out of desperation or in search of support — offer early insight into how the therapy is being experienced outside the tightly controlled clinical trial setting. But the leadership at Sarepta has not even acknowledged this outpouring of information. Clearly, the system is broken.

Looking ahead, I believe the next generation of gene therapies will bring more promise, more safety, and better efficacy. We will have better vectors, and sponsors will provide broader data. I firmly believe accelerated approval for drugs that treat rare diseases is essential, and that those drugs should use this pathway to bring therapies to patients and families as quickly as possible — and I don’t believe that needs to be years away.

But to make that vision happen, and to ensure families feel equipped to make hard decisions, we need to see changes.

That means mandatory confirmatory studies that collect real-world evidence. In addition, the sponsor and policymakers need to take responsibility to ensure all data, good or bad, is entered into the FAERS system accurately and completely — or else they must create a better system. The system should push that information proactively to our physicians, rather than requiring them to look it up themselves. The FAERS dashboard should be a mandatory resource, and there should be timely and accurate documentation — not haphazard data entry with no accountability. Irreversible treatments require a central, public repository of adverse events, interventions, and easy-to-understand multidisciplinary-guided access to complex multisystem protocols for when things do not go as planned.

We need multidisciplinary protocols with input from hepatologists, nephrologists, transplant experts, and those who have had prior experience with gene therapy. Furthermore, our neuromuscular doctors need to be supported fully. They were not necessarily trained in acute liver failure; they are not transplant physicians. They are the experts we count on daily for care of Duchenne — not liver disease.

We need FDA rigor that does not slow innovation but ensures a system with easy-to-understand informed consent and mandated post-market follow-through. And if there is failure, there need to be consequences. Regulators must require better transparency and tighten safety oversight. Policymakers must ensure that those who say yes to clinical science and rely on an “approved” commercial drug are honored with more than thoughts and prayers. They deserve action and reform.

Our community deserves these higher standards, and it is the sponsors who must meet them with partnership — not dictatorship — with our community.

Elevidys’ current label does not reflect the severity of what is happening. There should be a black box or at least a more rigorous safety information on the label so physicians know how to treat these patients.

Families are mourning. Those who said yes are terrified. We all are. The most respectful tribute is to fix this system — worthy of the courage our families have shown.

Those families, and those no longer here, deserve a drug development system that matches their bravery with integrity.

Understanding Aluminum Toxicity: What You Need to Know

Thu, 05 Jun 2025 13:13:45 GMT

Aluminum is the third most abundant element in the Earth's crust and has a wide range of industrial and household applications. However, increasing evidence suggests that aluminum exposure may pose health risks, particularly concerning brain health and toxicity. In this article, we’ll explore what aluminum is, how it affects our bodies—especially the brain—and how it may contribute to neurodegenerative diseases like Alzheimer’s. We’ll also discuss sources of aluminum exposure, methods of testing for toxicity, and natural approaches to detoxification.

What Is Aluminum?

Aluminum (Al) is a lightweight, silvery-white metal commonly used in manufacturing due to its durability and resistance to corrosion. It’s found in everything from packaging materials and construction to consumer products such as cookware, deodorants, and vaccines. While aluminum is generally considered safe in limited amounts, accumulating evidence points to potential health concerns with chronic exposure.

How Does Aluminum Get Into Our Bodies?

1. Dietary Sources

Processed foods: Aluminum-containing additives (e.g., anti-caking agents, coloring agents)
Baking powders and processed snacks

2. Household Items

Aluminum foil: Used in cooking and food storage
Nonstick cookware: Some nonstick coatings contain aluminum
Deodorants: Aluminum compounds like aluminum chloride are common active ingredients

3. Vaccines

Some vaccines contain aluminum salts (adjuvants) to boost immune response. While considered safe by regulatory agencies, concerns have been raised about long-term accumulation, especially in vulnerable populations.

4. Personal Care Products

Antiperspirants containing aluminum compounds

5. Environmental Exposure

Air pollution from industrial emissions
Drinking water contaminated with aluminum

Once ingested or absorbed, aluminum can accumulate in tissues, including the brain, bones, and lungs.

Aluminum's Impact on the Brain and Neurodegeneration

Research indicates that aluminum can cross the blood-brain barrier, accumulating in neural tissues. Studies have linked aluminum exposure to neurodegenerative conditions, most notably:

Alzheimer’s Disease: Some studies suggest that aluminum may promote amyloid plaque formation, oxidative stress, and neuroinflammation—hallmarks of Alzheimer’s pathology.
*Other neurodegenerative disorders: Parkinson’s disease and multiple sclerosis have also been associated with metal imbalances, including aluminum. Autism likely has a link to Aluminum and other heavy metals as well

While causality remains under investigation, the potential neurotoxicity of aluminum warrants caution, especially in individuals with existing neurological vulnerabilities.

Aluminum and Heavy Metal Toxicity: Inflammation and Systemic Effects

Heavy metals like aluminum can trigger systemic inflammation, disrupting normal cellular functions. Aluminum may activate immune responses, leading to chronic inflammation, which is a common pathway implicated in many chronic diseases, including cardiovascular disease, autoimmune disorders, and neurodegeneration.

Chronic inflammation caused by aluminum accumulation can damage tissues and impair detoxification pathways, exacerbating health issues over time.

Testing for Aluminum Toxicity

1. Hair Analysis

Pros: Non-invasive, reflects long-term exposure
Cons: Variability due to external contamination, less standardized

2. Blood Tests

Pros: Measures recent exposure, widely available
Cons: Aluminum has a short half-life in blood; levels may not reflect total body burden

3. Post-Chelation Urine Testing

Method: Administering a chelating agent (e.g., EDTA) followed by urine collection to assess metal mobilization
Pros: Indicates body burden of metals
Cons: Requires medical supervision, may cause side effects

Summary:

Hair analysis is useful for assessing long-term exposure but can be contaminated.
Blood testing provides a snapshot of recent exposure.
Post-chelation urine testing-gives a measure of body burden but involves intervention.

Natural Approaches to Removing Aluminum from the Body

A functional medicine approach emphasizes supporting the body’s natural detoxification pathways:

1. Sauna Therapy (Sweat Induction)

Purpose: Promotes excretion of heavy metals via sweat
Implementation: Infrared or traditional saunas, starting with short sessions

2. Binding Agents*

Examples: Modified citrus pectin, chlorella, zeolite, cilantro
Mechanism: Bind metals in the GI tract to reduce absorption and facilitate elimination

3. Supporting Liver and Kidney Function

Ensuring optimal function of detox organs enhances metal clearance

4. Nutritional Support

Adequate intake of antioxidants (vitamins C and E), glutathione precursors, and minerals like magnesium and selenium to combat oxidative stress

5. Chelation Therapy

Medical chelators (e.g., EDTA, DMSA) can be used under supervision to remove heavy metals, but should be approached cautiously due to potential side effects

References

1. **Exley, C. (2013).** *Aluminum and Alzheimer's disease: the science that describes the link.* Journal of Alzheimer's Disease, 33(2), 377–387.

2. **Krewski, D., et al. (2007).** *Human health risk assessment for aluminum, including exposure from vaccines.* Regulatory Toxicology and Pharmacology, 49(3), 258-262.

3. **Krewski, D., et al. (2010).** *Human health risk assessment for aluminum, including exposure from vaccines.* Toxicology and Applied Pharmacology, 254(3), 262-275.

4. **Genuis, SJ. (2011).** *Heavy metals and human health: current evidence and future directions.* Alternative Medicine Review, 16(4), 363–371.

5. **Genuis, SJ., et al. (2014).** *Heavy metal detoxification: a review.* Alternative Medicine Review, 19(2), 124-139.

6. **Kostoglou-Antetrou, K., et al. (2008).** *Aluminum and neurodegeneration: an update.* Journal of Clinical Neuroscience, 15(5), 558–561.

JAR of Hope Named 2025 Non-Profit of the Year by Monmouth Regional Chamber of Commerce

Wed, 04 Jun 2025 13:15:26 GMT

We are incredibly honored to share that JAR of Hope has been named the 2025 Non-Profit of the Year by the Monmouth Regional Chamber of Commerce as part of their prestigious Beacon Awards .

The Beacon Awards are designed to recognize organizations that exemplify outstanding community enrichment through service, leadership, and achievement. It means so much to us that both the MRCC membership and the community at large recognized the work we do and the mission we live by every day.

This award is not just a recognition of our team’s efforts—it’s a reflection of the amazing community that supports us, believes in our cause, and stands with us in the fight to raise awareness, fund research, and bring hope to families affected by Duchenne Muscular Dystrophy.

Thank you to the Monmouth Regional Chamber of Commerce, to those who nominated us, and to everyone who continues to walk this journey with us. We are deeply grateful—and more motivated than ever to keep pushing forward.

— The JAR of Hope Team

The Microbiome and Its Impact on Autism: Understanding the Gut-Brain Connection

Tue, 22 Apr 2025 14:39:35 GMT

As research into autism spectrum disorder (ASD) continues to evolve, one intriguing area of study is the role of the microbiome—the trillions of microorganisms living in our guts. Emerging evidence suggests that the health of our microbiome may have significant implications for children with autism, particularly in relation to gut disorders and neurological development. This blog explores the connection between the microbiome and autism, emphasizing the importance of detailed stool analyses, checking for parasites, and understanding the gut-brain relationship.

What is the Microbiome?

The microbiome refers to the vast community of bacteria, viruses, fungi, and other microorganisms that inhabit our digestive systems. These microorganisms play a crucial role in digestion, immunity, and even brain function. In children with autism, studies have indicated that there may be an imbalance in the microbiome, often referred to as dysbiosis. This condition can lead to gastrointestinal issues that are commonly reported in children with ASD, such as constipation, diarrhea, and abdominal pain.

Importance of Detailed Stool Analysis

A detailed stool analysis is a valuable tool for understanding the microbiome's composition and function. By examining stool samples, healthcare providers can identify bacterial imbalances, inflammation markers, and other indicators of gut health. This analysis can reveal whether a child is experiencing dysbiosis, which may contribute to their autism-related symptoms.

For instance, a stool test might show an overgrowth of harmful bacteria or a deficiency of beneficial ones. Identifying these imbalances can guide dietary and therapeutic interventions aimed at restoring gut health. Parents of children with autism are encouraged to discuss stool analysis with their healthcare provider as part of a comprehensive approach to treatment.

Checking for Parasites

Another crucial aspect of gut health is the presence of parasites. Many children with autism may have undiagnosed parasitic infections that can further complicate their gastrointestinal symptoms and overall well-being. Common parasites include Giardia, Blastocystis, and various types of worms. These organisms can disrupt the gut microbiome, leading to inflammation and digestive issues.

Testing for parasites through stool analysis is essential, as treating these infections can significantly improve gut health and potentially alleviate some autism-related symptoms. Parents should be vigilant about gastrointestinal symptoms in their children and consider discussing parasite screening with their healthcare provider

Leaky Gut and Its Implications

One condition often associated with dysbiosis is "leaky gut," where the intestinal lining becomes more permeable than normal. This permeability allows toxins and undigested food particles to enter the bloodstream, triggering inflammation and immune responses. In children with autism, leaky gut may contribute to behavioral and neurological symptoms.

Conclusion

The relationship between the microbiome and autism is a complex and rapidly evolving field of study. Understanding the importance of gut health, conducting detailed stool analysis, checking for parasites, and recognizing the implications of leaky gut can be vital steps for parents seeking to support their children with autism. While more research is needed, there is growing hope that addressing gut health may lead to improved outcomes for children with ASD. As always, it’s essential for parents to work closely with healthcare professionals to develop tailored strategies that best support their child's unique needs.

By prioritizing gut health, we may unlock new avenues for understanding and improving the lives of children with autism, paving the way for a brighter future.

Researchers Find 102 Genes Linked to Autism in One of the Largest Studies of Its Kind to Date

Thu, 10 Apr 2025 18:14:13 GMT

A groundbreaking study published in Cell on January 23, 2020, has identified 102 genes associated with autism spectrum disorder (ASD), including 30 previously unlinked to the condition. Led by Joseph Buxbaum, director of the Seaver Autism Center for Research and Treatment at Mount Sinai, the research analyzed over 35,000 DNA samples, nearly 12,000 of which were from individuals with autism.

The study leveraged advanced genetic sequencing technologies and data from the Autism Sequencing Consortium, an international collaboration of scientists sharing samples and data since 2010. By comparing genetic sequences from individuals with autism to their siblings and to unaffected individuals, the researchers identified both inherited mutations and spontaneous (de novo) genetic alterations that may contribute to ASD.

Notably, the research distinguished genes more closely associated with autism from those linked to other neurodevelopmental disorders, such as intellectual and motor disabilities. This differentiation enhances our understanding of autism's molecular mechanisms and could inform the development of targeted treatments.

Dr. Geraldine Dawson, director of the Center for Autism and Brain Development at Duke University, hailed the study as "one of the most important, comprehensive studies on the genetics of autism to date," emphasizing its role in elucidating the complex genetic landscape of ASD.

This research represents a significant step forward in autism genetics, offering hope for more personalized and effective interventions in the future.

January 24, 2020 2:19 PM EST

Time Magazine

CBD improves behavior of kids with autism — with new studies testing children as young as 5

Thu, 10 Apr 2025 18:11:46 GMT

A new study is shedding light on the potential of CBD to ease behavioral challenges in children with autism. Researchers in Israel found that cannabidiol (CBD), a non-psychoactive compound derived from cannabis, significantly improved symptoms like anxiety, aggression, and restlessness in kids on the spectrum.

The peer-reviewed study, published in the journal Translational Psychiatry, followed 82 children and teens with autism who were treated with CBD-rich cannabis oil for six months. Results showed that many experienced meaningful improvements in behavior and social communication — with minimal side effects.

Parents reported reduced irritability, fewer outbursts, and better sleep among participants. One mother shared that her nonverbal son began using more words and expressing emotions for the first time.

While researchers emphasized that CBD isn’t a cure for autism, they believe it could be a safe and effective option to support children with challenging symptoms — especially when traditional treatments fall short.

As interest in alternative therapies grows, this study adds to the growing body of research suggesting that CBD may offer a new avenue of hope for families navigating autism.

Published April 8, 2025, 9:46 a.m. ET

New York Post

Children with autism are showing ‘dramatic improvement’ with this cheap drug — and doctor behind its research says it may one day prevent the disorder

Thu, 10 Apr 2025 18:08:54 GMT

As autism diagnoses continue to rise in the U.S., scientists are exploring innovative treatments — and one unexpected option may be a generic cancer drug. Leucovorin, typically used to reduce the side effects of chemotherapy, is showing remarkable promise in improving symptoms of autism.

Over the past 13 years, Dr. Richard Frye, a pediatric neurologist, has gathered compelling evidence that leucovorin may help children with autism — including nonverbal kids who began speaking after treatment. While there’s no single “autism pill,” Frye believes leucovorin offers real hope for some families.

The drug’s potential lies in its ability to bypass a condition called cerebral folate deficiency (CFD), which is linked to low levels of folate in the brain. Many children with autism have been found to carry folate receptor autoantibodies that block the brain’s folate transport. Leucovorin, a folic acid derivative, can restore folate levels despite this blockage.

Frye’s early patients often showed dramatic improvements — from reduced seizures to increased communication and social interaction.

With autism now affecting about 1 in 36 children in the U.S., the need for effective treatments has never been greater. While further research is needed, leucovorin is offering a surprising and hopeful path forward.

Published March 19, 2025, 6:00 a.m. ET

New York Post

Understanding Autism: A Biomedical Approach to Training and Treatment

Mon, 07 Apr 2025 14:51:27 GMT

Autism Spectrum Disorder (ASD) is a complex neurodevelopmental condition that affects communication, behavior, and social interaction. As parents of autistic children, you may often find yourself navigating a maze of information, therapies, and treatment options. One approach that has gained traction in recent years is the biomedical model, which posits that by modifying certain environmental factors, we can help improve symptoms and enhance the quality of life for autistic individuals.

The Genetic Component of Autism

Autism is believed to have a genetic basis, with studies indicating that certain genetic factors may predispose individuals to develop the condition. However, it’s crucial to understand that not all genetic components are set in stone. Some genetic expressions can be influenced by environmental factors, meaning that changes in the environment can potentially modify how these genes function.

One such gene of interest is the **MTHFR gene**. To simplify, the MTHFR gene is responsible for producing an enzyme that helps process amino acids—the building blocks of proteins—and is essential for the body's ability to use folate (a type of B vitamin). This gene plays a significant role in the methylation process, which is involved in regulating many bodily functions, including DNA repair and detoxification.

When the MTHFR gene is not functioning optimally—often due to mutations—this can lead to a variety of health issues, including problems with metabolism and mental health. In the context of autism, some researchers believe that supporting the body's methylation process through dietary changes and supplements can help address certain symptoms associated with the disorder.

The Role of Environment in Modifying Autism

The biomedical approach emphasizes the idea that while some genetic factors may be permanent, many are modifiable through environmental changes. This could include dietary adjustments, lifestyle modifications, and the use of specific supplements aimed at enhancing overall health and well-being.

For instance, research has shown that certain dietary interventions, such as gluten-free or casein-free diets, can lead to improvements in behavior and communication skills in some children with autism. Furthermore, ensuring adequate intake of vitamins and minerals, particularly those involved in the methylation process, can support brain health and function.

A study published in *The Journal of Autism and Developmental Disorders* found that children with autism who were supplemented with methylcobalamin (a form of vitamin B12) showed improvements in communication and social skills. This illustrates the potential impact of targeted nutritional support in managing autism symptoms.

The Mind-Body Connection

Another aspect of the biomedical approach is addressing the mind-body connection. Many doctors and scientists argue that by focusing on mental health and emotional well-being, we can facilitate better outcomes for autistic individuals. Stress, anxiety, and other emotional factors can exacerbate the challenges faced by children with autism. Therefore, therapies that promote mental wellness—such as cognitive-behavioral therapy, mindfulness, and relaxation techniques—can play a vital role in treatment.

Furthermore, some researchers are investigating how gut health may influence autism symptoms. The gut-brain connection is increasingly recognized in modern medicine, and studies suggest that a healthy gut microbiome may positively impact behavior and cognitive function. This underscores the importance of a holistic approach that considers both physical and mental health.

Conclusion

While autism has genetic components that may be permanent, the biomedical approach offers hope for parents seeking to improve their child's condition through modifications in environment and lifestyle. By understanding the role of genes like MTHFR and the impact of nutrition and mental health, we can empower ourselves to make informed decisions about treatment options.

As research continues to evolve, staying informed and connected with healthcare professionals who specialize in autism is essential. Remember, every child is unique, and what works for one may not work for another. Nevertheless, with the right support and interventions, the journey toward improved well-being for autistic individuals can be a positive and transformative experience.

If you’re interested in exploring more about the biomedical approach to autism, consider connecting with professionals or support groups that focus on this area. Together, we can create a brighter future for our children.

Sarepta's 27% Sell-Off Is 'Overblown,' 'Overdone' And 'Overly Bearish.' Here's Why It Happened.

Wed, 19 Mar 2025 14:07:43 GMT

From Investors.com

Sarepta stock crashed Tuesday after a patient who received its approved gene therapy, Elevidys, died due to acute liver failure.

Elevidys treats Duchenne muscular dystrophy, a devastating disorder that causes progressive muscle deterioration. According to Sarepta Therapeutics ( SRPT ), the patient developed acute liver failure following treatment with Elevidys. He also had a recent cytomegalovirus infection, or CMV, which could have been a contributing factor. CMV can injure the liver.

This is the "event people had feared," said RBC Capital Markets analyst Brian Abrahams. But it "shouldn't spell the end for Elevidys."

"The news is certainly not good, but given the known liver toxicities associated with Elevidys and all gene therapies, the fragile nature of the patients with this deadly illness being treated and the growing use of the agent, it is perhaps not altogether surprising that a fatal (adverse event) could potentially be observed occasionally," he said in a note.

Still on today's stock market , Sarepta stock toppled 27.4%, closing at 73.54.

'Very Unique' Case

Sarepta didn't provide any identifying characteristics about the patient other than to describe him as a "young man."

William Blair analyst Sami Corwin said the patient was a 16-year-old boy. Analysts noted older and non-ambulatory patients would typically weigh more and require a larger dose of Elevidys. Further, older patients have a higher morbidity, Leerink Partners analyst Joseph Schwartz said in a report.

This is the first fatality among Elevidys recipients. Sarepta says it has treated north of 800 patients weighing up to 300 pounds with Elevidys in testing and following approval. Sarepta believes this case is "very unique," Schwartz said.

"Although we acknowledge that such severe side effects associated with mortality can certainly be alarming and cause the community to question the risk/benefit of treating older patients, we believe that the very low overall incidence which we estimate at less than 0.125% based on aggregate exposure to date is encouraging," he said.

He reiterated his outperform rating on Sarepta stock.

Well-Known Side Effect

Liver toxicity is associated with AAV-based gene therapies. These drugs use non-harmful viruses known as adeno-associated viruses, or AAV, to deliver therapeutic genes to the cells. In this case, the drug tells the body how to make a shortened version of the dystrophin protein that's missing in Duchenne patients. Dystrophin helps keep muscles intact and functional.

William Blair's Corwin noted two patients died following treatment with Novartis ' ( NVS ) Zolgensma, a treatment for spinal muscular atrophy.

"Overall, we see this event as unlikely to affect patient/physician interest in Elevidys in the near term or Sarepta's full year 2025 product revenue guidance of $2.9 billion to $3.1 billion," she said in a report.

Analysts called the sell-off "overblown," "overdone" and "overly bearish."

"Thus we think this is an overreaction and presents a buying opportunity ahead of a continued strong Elevidys launch," Leerink's Schwartz said.

Meanwhile, shares of Regenxbio ( RGNX ), which is working on Duchenne gene therapies, rose 11.6% to 7.81. Solid Bio ( SLDB ), another gene therapy competitor, saw shares shrink 4.9%, trading down at 5.10.

Transforming the Future of Duchenne Treatment: A Partnership for Progress

Tue, 10 Dec 2024 16:27:15 GMT

JAR of Hope is proud to share the groundbreaking partnership between Roche and Sarepta Therapeutics, aimed at reshaping the way Duchenne muscular dystrophy (DMD) is treated.

Duchenne is a rare genetic disease that progressively weakens muscles, affecting movement, breathing, and heart function. It predominantly impacts boys, with approximately 1 in 3,500–5,000 boys born worldwide being affected. The condition results from mutations in the DMD gene , which hinder the production of dystrophin, a vital muscle protein. Without dystrophin, muscles are more prone to damage, leading to a decline in function over time.

Currently, treatment options are limited, offering only modest benefits. However, advancements in genetic medicines and emerging gene therapies provide hope for a future where Duchenne patients can live longer, healthier lives. These therapies aim to restore dystrophin production, offering a potential breakthrough for a wider range of patients.

Through this collaboration, Roche, Sarepta Therapeutics, and the Duchenne community are driving innovation and advocacy to address the urgent need for new, effective treatments. Together, we are working to create a brighter future for those affected by Duchenne muscular dystrophy.

To read more about this advancement, please click here .

For updates on our mission and how you can support our fight, visit JAR of Hope .

JAR of Hope Changes a Family's Life

Tue, 06 Aug 2024 14:25:53 GMT

Ashley and Adam Wells live in a small town in upstate NY, with their two children, Adam Jr, 13, and Evelyn, 8.

Adam Jr. was diagnosed with Duchenne muscular dystrophy just after his sixth birthday. And needless to say, life hasn’t been the same for the Wells family since then.

“It all started when Adam’s teacher mentioned to me that his calves seemed unusually large,” Ashley Wells says. “One thing led to another…actually, one test led to another. Eventually he was diagnosed with Duchenne muscular dystrophy. And, like most people, we had never heard of this disease.” (Adam Jr. isn’t alone, though; Ashley notes there are now four kids in their school district with Duchenne.)

Duchenne md, of course, is a progressive muscle-wasting disease, with no cure even after more than 200 years. It occurs mostly in boys. By nine or ten, these kids start losing the ability to walk. By the mid-teens, they’re generally breathing on ventilators. And their lifespans extend generally only to the mid-twenties.

In recent years, Adam Jr’s condition has made it harder for the Wells family to go out on the hiking, camping, and fishing trips they love. Automobiles, of course, aren’t made with Duchenne patients in mind. And it’s been getting progressively more difficult to get Adam Jr. into the family car, then out of the car and into the wilderness, and then back into the car again.

“Adam loves school; he’s a straight-A student,” Ashley says. “But he loves Nature just as much. And it was getting harder and harder to get him out there, because our car – almost any car, really – isn’t built for transporting kids with Duchenne.”

Then Lady Luck stepped in. Ashley’s sister-in-law did some research, and found out about JAR of Hope. So she contacted JOH, and after speaking with Jim Raffone, Founder/CEO, she referred Ashley to him.

As the family had previously been contacted about Adam Jr. by several “schemers,” Ashley says, they wanted to check JAR of Hope out carefully. And that’s exactly what they did.

“The diagnosis of Duchenne md is shattering for families,” she says. “Needless to say, when you look for help, you want to be sure you can trust who you’re dealing with. So we took a careful look at JAR of Hope. Not only were they raising funds to research a cure for this disease, but they were doing it in unique ways. And I discovered they were really good at getting the word out about this horrible disease and its effect on families.”

There’s a good reason JAR of Hope is so good at getting the word out. Jim and Karen Raffone’s youngest child, 15-year-old James Anthony (”Jamesy”), also has Duchenne. So they know firsthand the pain parents of these kids feel.

The average charitable foundation doesn’t have a team that climbs Mount Everest to raise funds for a cure, which JOH did two years ago. Or that participates in athletic events all over the world, some of them lasting a full week, and involving freezing nighttime temperatures in the mountains or steaming daytime temperatures in the desert. (And the average charitable foundation doesn’t have a founder like Jim Raffone, who, at a muscular 53, leads the JOH team in all these events.)

Ashley Wells remembers a conversation in which she mentioned to Jim the difficulty in getting Adam Jr. into and out of the car to enjoy the outdoor pursuits he loves.

“We did some research into the type of car that would be appropriate,” Jim Raffone says, “and that would also be available. And when we found such a car, we went after it.”

What they found was a 2019 Dodge Grand Caravan. But not just any 2019 Dodge Grand Caravan.

The car seemed perfect for the Wells family. It could go off-road and even onto beaches. It had a fishing-rod holder, and a traction chair. It also had a joystick, which allows people with motor disabilities to operate the steering wheel by remote control. And it had a lot of room.

But a car like that costs a lot of money. Some of it was raised by a benefit in the town. Some of it was also raised by administrators and teachers at Adam’s school. However, by the end of these efforts, the family was still nearly $8,000 short.

“That’s when we upped our involvement,” Jim Raffone says. “We raised the rest of the funds for the family. And now they have a car that Adam Jr. can get in and out of comfortably – with futuristic electronics that really help him – so he can experience all the great outdoors pursuits he loves.”

Like most parents with kids with Duchenne md, Ashley Wells will never forget when she heard the diagnosis.

“I’d never heard of this disease until that day,” she says. “I received a phone call at work from the doctor…and it was the hardest thing I’ve ever had to hear. But we just keep pushing forward.

“And having an ally like JAR of Hope gives us real hope.”

A Familiar Name Joins the JOH Roster: Kimberly Buk

Tue, 06 Aug 2024 14:21:44 GMT

Kimberly Buk (formerly Kravitz) is a two-time Emmy Award-winning television journalist with a long string of notable accomplishments and awards. She’s a familiar face in the Tri-State area, in both print and electronic media.

In 2017, she was the producer of a program called “Jersey Matters,” working with well-known host/interviewer Larry Mendte. And, after learning about Jim Raffone, who’d started up JAR of Hope a few years previously, she had Jim on the program for an interview.

The interview went well, and obviously was a very touching event for Kimberly. During it, she learned that Duchenne muscular dystrophy is a fatal, muscle-wasting childhood disease that still has no cure – even after more than 200 years. She learned, too, that Duchenne md occurs only once in every 3,500 live births, mostly in boys. And she learned that there are only about 17,000 kids in America with this disease.

“When I learned a little about Jim and Karen and their son James Anthony (“Jamesy”), who has Duchenne muscular dystrophy,” Kimberly says, “I learned that this little-known disease was killing children before they had a chance to become adults.”

Jim Raffone explained on the show that kids with Duchenne start losing the ability to walk as early as nine or ten. Start losing the ability to breathe on their own by 12 or 13. And generally pass on - from asphyxiation - in their early-twenties. And he explained, too, that when he and Karen asked the doctors who had diagnosed Jamesy what they could do to fight this disease, the doctors said simply, “There is no cure. Just take him home and love him until he passes on.”

Kimberly was struck by the power of those words to deprive parents of any hope at all. But she learned quickly that the Raffones didn’t indulge in self-pity - or in entertaining the thought of giving up.

“As Jim spoke to Larry on the show,” she says, “I kept thinking how powerful his words were, and how touching they were. I wondered how anyone could not be touched by what he was saying. And how anyone could not be interested in helping this family – and the other 17,000 Duchenne families across the U.S. – to fund research for a cure for these children.”

As a result, Kimberly began helping JAR of Hope out as a media liaison. She and Jim also founded an organization called the Leadership Executive Alliance, of which she is now President.

“It gives me a chance to become a sort of Brand Ambassador for JAR of Hope,” she says. “And to help with the events that they put on to raise funds for a cure.” (The University of Florida has already offered to conduct research for a cure into Duchenne muscular dystrophy…if JAR of Hope can raise the $750,000 necessary to fund the research.)

Kimberly Buk notes, as well, that Jamesy Raffone is now 15 years old, and lost the ability to walk several years ago. For all these kids, this is truly a race against time.

JAR of Hope is a tremendous community asset. Jim Raffone, who’s now 53, has traveled widely around the world to participate in athletic endeavors to raise money to save these kids. And among his accomplishments are winning both of the professional boxing matches in which he’s participated, running in numerous marathons, conquering “Ultra” athletic events such as the tortuous Alps-to-Ocean In New Zealand, 300-mile runs through several states…and two years ago, leading a team from JAR of Hope in climbing to the highest base-camp on Mount Everest, at, 18,372’.

“When Jim and I worked on a new podcast together,” Kimberly says, “we also started thinking of other new ways to work together. We live in the same community. We know each other’s families. And I’m doing the best I can to use my broadcasting skill-set to help JAR of Hope get the word out.”

She also notes that as JAR of Hope grows in the Tri-State region, her goals don’t stop there. She says events such as the upcoming (Oct. 6) New Jersey Run 4 Hope Half-Marathon help spread the word about Duchenne and its effects on local families. And about all the good being done by JAR of Hope. (For information on this event: https://www.jarofhope.org/ ; (732) 414-6670; https:// The Race|NJ Run4Hope .com

“We’d like to help take JAR of Hope national,” Kimberly notes. (Note: JAR of Hope has recently expanded into Europe, and has plans for future expansion there.) “We’d like to expand our alliances, and to grow teams around the country.”

She notes there are plenty of web content and media opportunities for events, and that her broadcasting skill-sets put her in a unique position to help.

“I feel very passionate about trying to save Jamesy’s life, and the lives of all the other kids with Duchenne,” Kimberly Buk says. “The struggles of the Raffone family are near and dear to my heart. And the values of supporting these families and of charitable endeavors align with my morals.

“So I’ll be there every step of the way.”

Assistance for Families with Autism

Wed, 17 Jul 2024 15:58:38 GMT

Office of Autism

The NJ Department of Human Services’ (DHS) Division of Developmental Disabilities ( DDD ) has been the lead resource agency for individuals with developmental disabilities and families to seek programs and services since 1959. The Division has a broad scope – addressing individual diagnoses that fall collectively within the definition of intellectual and developmental disabilities. Categories and eligibility information can be found here. The Division's mission to protect the health and safety of the individuals it serves, promote equity and fairness among diagnoses and expend responsibly and efficiently state tax dollars continues, today.

The most recent study by the U.S. Department of Health and Human Services’ Centers for Disease Control and Prevention identified the national Autism Spectrum Disorders (ASD) rate as 1 in 54 births. New Jersey's rate is reportedly 1 in 32. As such, the state has taken a number of significant steps to be responsive to issues associated with its population of individuals with ASD.

An Adults with Autism Task Force (AWATF) was created by statute and charged with making recommendations to the Governor and Legislature about programs and services that would meet the needs of adults with ASD. The report was released in late 2009. Acting upon one of the Task Force’s recommendations, DHS established an Office of Autism (OOA) under the DDD umbrella in August 2010. The Office will provide a centralized place for DDD staff to focus on issues associated with ASD, identify and work with staff in sister state agencies to develop plans of service coordination for persons with ASD and promote, disseminate and organize best practices in the training of staff and other supports to people with ASD.

To visit their website, please visit: https://www.nj.gov/humanservices/ddd/individuals/autism/

Breakthroughs in Stem Cell Research: How Mesenchymal Stem Cells (MSCs) Combat Duchenne Muscular Dystrophy

Wed, 17 Jul 2024 15:55:12 GMT

Duchenne Muscular Dystrophy (DMD) is a devastating genetic disorder characterized by progressive muscle degeneration and weakness. Affecting approximately 1 in every 3,500 to 5,000 male births worldwide, DMD is caused by mutations in the dystrophin gene, leading to the absence of dystrophin, a crucial protein for muscle integrity. As research advances, mesenchymal stem cells (MSCs) have emerged as a promising therapeutic option, offering hope for those affected by this relentless disease.

Understanding MSCs

Mesenchymal stem cells are multipotent stromal cells capable of differentiating into various cell types, including muscle, bone, and cartilage cells. MSCs can be harvested from various sources, such as bone marrow, adipose tissue, and umbilical cord blood. Their unique properties, including immunomodulatory effects, trophic support, and differentiation potential, make them ideal candidates for regenerative medicine.

MSCs in the Fight Against DMD

1. Muscle Regeneration: MSCs have the ability to differentiate into muscle cells, potentially replacing damaged or missing dystrophin-expressing cells in DMD patients. This can lead to improved muscle function and slowed disease progression.

2. Anti-Inflammatory Effects: DMD is often accompanied by chronic inflammation, exacerbating muscle damage. MSCs secrete anti-inflammatory cytokines that can modulate the immune response, reducing inflammation and protecting muscle tissue from further damage.

3. Paracrine Signaling: Beyond cell replacement, MSCs secrete bioactive molecules that promote tissue repair and regeneration. These molecules can enhance the survival and function of existing muscle cells, support the formation of new blood vessels, and stimulate endogenous repair mechanisms.

Clinical Trials and Promising Results

Several clinical trials are underway to evaluate the safety and efficacy of MSC-based therapies for DMD. Early-phase trials have shown promising results, with patients exhibiting improved muscle strength, reduced fibrosis, and enhanced quality of life. Researchers are also exploring various delivery methods, such as systemic infusion and direct intramuscular injection, to optimize the therapeutic effects of MSCs.

Challenges and Future Directions

While MSC therapy holds significant promise, several challenges remain. These include ensuring consistent and scalable cell production, preventing immune rejection, and maximizing the cells' engraftment and survival in the hostile environment of dystrophic muscle. Ongoing research aims to address these hurdles, with advancements in genetic engineering, biomaterials, and combination therapies enhancing the potential of MSCs to treat DMD.

Conclusion

The application of mesenchymal stem cells in the treatment of Duchenne Muscular Dystrophy represents a beacon of hope for patients and families affected by this debilitating condition. With continued research and clinical development, MSCs have the potential to transform the landscape of DMD therapy, offering a new avenue for muscle regeneration and improved patient outcomes. The scientific community remains steadfast in its commitment to unlocking the full potential of MSCs, moving closer to a future where Duchenne Muscular Dystrophy is no longer a life-limiting diagnosis.

Pfizer Update on Clinical Trial

Fri, 10 May 2024 13:05:24 GMT

May 7, 2024

We are deeply saddened to share that a patient participating in our Phase 2 DAYLIGHT study for Duchenne muscular dystrophy has passed away suddenly. On behalf of everyone at Pfizer, we extend our sympathies to his family, friends, and those closest to his care.

We do not yet have complete information and are actively working with the trial site investigator to understand what happened. The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023.

Initial dosing for the placebo-controlled, randomized Phase 3 CIFFREO trial, which utilizes a crossover trial design, was also completed in 2023. We have decided to pause dosing associated with the cross-over portion of CIFFREO, and we are working with regulators and the independent external Data Monitoring Committee as we learn more about this event. Other than this pause in dosing, trial activities are continuing as scheduled.

Both the DAYLIGHT and CIFFREO trials are investigating fordadistrogene movaparvovec in patients with DMD, in different age cohorts: DAYLIGHT in boys 2 years to less than 4 years of age, and CIFFREO in boys 4 to less than 8 years of age. Beyond CIFFREO, the dosing pause does not apply to other ongoing trials in the fordadistrogene movaparvovec program as dosing has been completed in those studies.

The safety and well-being of the patients in our clinical trials remains our top priority, and we are committed to sharing more information with the medical and patient community as soon as we can. We are also aware that many in the patient community are hopeful about the potential benefit of fordadistrogene movaparvovec for the treatment of DMD, and we will continue to collect data from our trials to evaluate its ability to address this disease.

As always, if you have questions please do not hesitate to reach out.

Sincerely,

The Pfizer DMD gene therapy team

'Time is against us': Half-marathon closing Route 9 to fund desperately needed drug trial

Tue, 30 Apr 2024 13:08:12 GMT

Over the past decade, in a quest to fight the deadly disease that ails his son, Manalapan’s Jim Raffone has trekked to Mount Everest base camp , built a record-setting Lego chain , appeared on a special-edition Father’s Day card , and rung the closing bell of the New York Stock Exchange.

Next up: Holding a half-marathon on Route 9.

It took several years, but Raffone has lined up approvals for the 13.1-mile race, dubbed “NJ Run 4 Hope,” which will pass through five municipalities on the morning of Sunday, Oct. 6. Most of the course is along Route 9 South, which will shut down and reopen gradually as the race unfolds. Proceeds will fund a clinical trial for Duchenne muscular dystrophy — a rare and fatal genetic disorder that causes progressive muscle weakness.

Jim’s son James (Jamesy) Raffone was diagnosed with Duchenne at age 4 . He’s now 15 and a freshman at Howell High School. The life expectancy for Duchenne patients is generally in the mid-20s.

“Right now time is against us, so with this happening and with the help of all the mayors, it’s been a godsend,” Jim Raffone said. “I can’t thank everyone enough.”

This is believed to be the first half-marathon or marathon taking place mostly along Route 9. The race will begin at All American Ford in Old Bridge and proceed south through Marlboro and Manalapan, with a loop through Freehold Borough before ending at iPlay America in Freehold Township.

“We’re going to have a huge celebration and a business expo at iPlay,” Raffone said.

JAR of Hope, the nonprofit the Raffone family launched in 2014, has been granted approval by the U.S. Food and Drug Administration to begin clinical trials on an investigational drug to treat Duchenne. The trials will be conducted by researchers at the University of Florida.

Raffone said he’s hoping to raise $1 million with a field of at least 1,000 runners. The clinical study is expected to cost about $2 million; JAR of Hope has been fundraising for it since the study was approved in 2021.

“I would be blown away if we could get 3,000 people (to race),” Raffone said. “We can change the course of this disease by funding a clinical trial with one race.”

For more information on NJ Run4Hope, visit www.njrun4hope.com , email info@njrun4hope.org or call 732-414-6670.

Jerry Carino is community columnist for the Asbury Park Press, focusing on the Jersey Shore’s interesting people, inspiring stories and pressing issues. Contact him at jcarino@gannettnj.com.

The Towncast – Jim shares his journey up Mt. Everset

Mon, 08 Apr 2024 14:37:04 GMT

Jim shares his journey from learning his only son has Duchenne Muscular Dystrophy to running 10 marathons including running over 300 miles from Massachusetts to New Jersey, doing push up around the world and climbing Mt Everest, all to bring awareness to and helping find a cure for this terminal disease.

JAR of Hope team climbs Mt. Everest to save the first child

Fri, 07 Apr 2023 13:22:37 GMT

Nebraska man joins fight against Duchenne muscular dystrophy

Article from News Channel Nebraska

Thursday, April 6th 2023, 5:56 PM CDT

AUBURN – A Nebraska man joined a Jar of Hope team to set a world’s record on Mt. Everest in hope of gaining the world’s attention about the deadly disease Duchenne muscular dystrophy.

It is a story of triumph under dire circumstances with an unsettled ending and no happy beginning.

Dillon Doeden, who grew up in Talmage, got involved with the Jar of Hope after his son was diagnosed with Duchenne at the age of two.

It was heartbreaking, but simple for Doeden to learn how the disease has been known to science for 200 years and how no child can say they survived.

It was harder to find out what Doeden could do for his son Connor.

Jim: “When Dillon and I came across each other on a Facebook group, the questions Dillon was asking were questions I was asking a few years prior and I felt his loneliness. I felt his desire to learn about this disease and what he could do for his son. I recall feeling that way when I didn’t have help. I couldn’t for the sake of leaving him out there being vulnerable allow no one there to respond to this man’s questions because all he wants to do is help his son.

I felt it was my right to be there for him because now he was the freshman in this disease community and I was now a senior. It was my responsibility to give back to him.”

Jim Raffone, the founder of Jar of Hope and producer of the documentary “Climb for a Cure”, provided an answer. Within a month, Doeden was on his way to Nepal for the 80-mile climb to the Mt. Everest base camp.

Doeden: “Jim has a line and the first time we spoke on the phone he said ‘I think you might be my kind of crazy.’ I think that about sums it up. I have no real technical climbing experience, but I will do anything for my son, so would Jim, and that’s what brought the two of us together from across the country and led us across the world to Nepal. I know he would do anything for his son and I will do the same and we will continue to do whatever we can do to fight Duchenne.”

The men, along with Joseph Russo and Matthew Scarfo, did 10 pushups at the highest elevation to set the record. A total of $200,000 was raised to raise awareness about Duchenne and fund a clinical trial in Florida.

The third documentary produced by Jar of Hope in nine years.

Jim: “The reason we use film it to teach everybody about Duchenne muscular dystrophy because before my son was diagnosed in 2013 I had never heard about the disease Duchenne. Usually everybody we come across they too don’t know about the disease and the deadly effects it has on children.”

Doeden: “It just puts into perspective the lengths we are willing to do to help our children.”

Doeden, who worked with the U.S. border patrol and served in Iraq with the U.S. Navy, intends to join Jar of Hope for a 171-mile hike across the Arizona desert in September.

Franklin Square kickboxing marathon raises money toward Duchenne muscular dystrophy research

Thu, 06 Apr 2023 15:09:48 GMT

Originally posted on Newsbreak

A kickboxing marathon in Franklin Square raised money and awareness for Duchenne muscular dystrophy.

CKO Kickboxing hosted Jar of Hope’s fundraiser to find a cure for the rare, fatal childhood disease.

The foundation was founded by Jim Raffone after his son James was diagnosed with Duchenne when he was 4.

The disease occurs only once in every 3,500 live births, mostly in boys. Most die of the disease in their early 20s.

Raffone saif seeing the support of so many strangers gives him hope.

“It really puts a lot of wind in my sails…because it gives me hope that my fighting is being heard and that we can make a change. We can make a difference in this world,” he said.

The kickboxing marathon already raised $10,000 that will go toward research to find a cure.

JAR of Hope presents National Pushup Campaign to all students

Wed, 29 Mar 2023 15:02:06 GMT

Will you accept this challenge Millstone Twp. & Upper Freehold Regional School Districts?

Originally posted on The Source

Last week, the JAR of Hope organization visited Upper Freehold Regional School District (UFRSD) and Millstone School District for assemblies at each of their respective three schools. JAR of Hope is a New Jersey based non-profit organization committed to finding a cure for Duchenne Muscular Dystrophy (DMD), a terminal genetic disorder characterized by progressive muscle degeneration and weakness. Jim Raffone, the co-founder of this organization, spoke to each student body about DMD with age appropriate messages. Jim’s son, James, has Duchenne Muscular Dystrophy.

Jim has traveled the world and even climbed Mt. Everest to raise awareness and funding with the hope of finding more effective treatments, and potentially a cure, for Duchenne. Aside from awareness, Jim has challenged our students to overcome fears and celebrate life when possible. His motto of “I can, I will, I must” was heard and chanted by all.

The UFRSD and Millstone Township Schools have joined together to support Jim on his mission as two of our students are affected by Duchenne and Jim is a resident of nearby Manalapan. Both districts will be taking part in the “Push-Up Challenge” to raise money to support a clinical trial for Duchenne at the University of Florida.

Recently, JAR of Hope hosted the Push Up Challenge at the Jackson Township School District. At that event, there were approximately 2,000 students and staff participated. Jackson challenged our two districts to exceed their participation. Let’s go, Millstone and UFRSD!

“Hands on the Floor – Duchenne No More” is a national initiative teaching elementary, middle and high schools about Duchenne Muscular Dystrophy and encourages students to start their own fight for a cure by raising awareness of the disease and money to support the JAR of Hope’s clinical trial.

Students in both districts were provided an envelope to take home where they can (if they/you wish) obtain sponsors for push-ups that will help raise money for Duchenne research. The fundraiser will end on Wednesday, March 29th. Envelopes can be sent to your child’s homeroom or the main office. The actual push up event will occur in the very near future.

Participation in this fundraiser is completely voluntary. It is hoped, however, that knowing how Duchenne Muscular Dystrophy affects some of our own makes this a meaningful cause to support.

Students in all of Jim’s presentations were respectful and engaged during the awareness presentations. It was heartwarming to see students cheer for their classmates with Duchenne.

“Jim’s passion and desire to find a cure for Duchenne was evident in every presentation,” UFRSD Superintendent Mark Guterl said. “His message of “I can, I will and I must” resonated with kids and staff. No obstacle is too big – a great message.”

Dr. Christopher Huss Superintendent of the Millstone Township School District added, “In Millstone Schools, we are continually looking for ways for our students to get involved in relevant and meaningful learning experiences that also benefit others. When the Jar of Hope campaign was presented to us, it was obvious we had to get involved as it fits with those goals perfectly.”

Millstone and Upper Freehold – will you accept this challenge and exceed Jackson’s participation in the JAR of Hope “Hands on the Floor – Duchenne No More” campaign?

Italfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy

Tue, 21 Feb 2023 16:13:52 GMT

-- Study meets primary endpoint with secondary and exploratory endpoints showing consistency with primary endpoint --

-- Data reinforces previously observed safety profile for Givinostat; treatment in boys with DMD continues to show a good tolerability profile --

-- The company is planning to discuss the potential for marketing application submission with regulatory authorities for Givinostat in DMD --

-- Results were presented at the Annual PPMD Conference on June 25, 2022 --

New Paragraph

Hyperbaric Oxygen Treatment (HBOT) Study

Tue, 24 Jan 2023 17:37:48 GMT

Hypoxia as a Modulator of Cardiac Disease in Duchenne muscular dystrophy

by Dr. DeWayne Townsend

Jamesy is one of the first children with Duchenne muscular dystrophy to undergo HBOT treatment. Through this study, his marked improvements have led to the first ever hyperbaric pre-clinical trial to be hosted by the University of Minnesota. The launch of the HBOT research is a primary focus of our current fundraising efforts. The findings may provide the vital information needed to prove the clinical efficacy of oxygen therapy, and could lay the groundwork to make this treatment available to all children suffering from Duchenne.

The goal of the HBOT study is to enable these children to walk for as long as possible, until a cure can be found.

SIGN A VIRTUAL PETITION FOR EQUAL JUSTICE FOR NJ CHARITIES

Tue, 24 Jan 2023 17:33:58 GMT

Equal justice for Charities: allow Charities to sell raffles tickets on-line in NJ

The body content of your post goes here. To edit this text, click on it and delete this default text and start typing your own or paste your own from a different source.

We need to change the laws stopping Charites from selling raffle tickets online.

You can: bet on a horse, baseball game, football game for profit but you CANNOT purchase a raffle ticket online through a charity website for a game of chance to help save a child’s life!!

New Jersey is the unquestionable leader when it comes to land-based gambling on the East Coast and has more legal gambling options than any other state. Residents and visitors of the Garden State can choose between dozens of live and online casinos and sports betting sites (as stated on the https://www.letsgambleusa.com/new-jersey/).

Most of the high-profile gambling establishments are located on the Atlantic City Boardwalk, but it’s worth remembering that New Jersey also has several pari-mutuel racetracks on the outside. The state allows charitable gambling and has its own lottery. New Jersey was the second state to regulate its intrastate online gambling market, which means that the local gambling enthusiasts are free to get some licensed casino and poker action without having to turn to offshore iGaming sites.

With all this said, NJ does not allow charities to sell raffle tickets online. Why? Because their statute was written in 1950 and the Internet did not exist then. Furthermore, the statute does not say a charity cannot sell raffle tickets online, It is the interpretation of the statute by the NJ AG’s office that prohibits charities from doing so.

I am asking you to sign this petition so we can change the law to allow all charities in the state of NJ to sell raffle tickets online. Given the circumstances we are navigating through and are currently in during this COVID pandemic, we are unable to gather together to raise money for causes near and dear to our hearts and for these individuals, our loved ones, who are most in need of our help at this time.

PLEASE SIGN THIS PETITION!

The solution is to change the statute. JAR of Hope has hired an attorney to help fight this in court and has enlisted the help of Congressman Chris Smith and Senator Thompson to help us make the change that is so gravely needed. However, your signatures are imperative to move this along in the process. Once this statute has been overturned, ALL Charities in the state of New Jersey will have the opportunity to raise funds through online raffle ticket sales, not just ours. This will be a victory for the thousands of charities that exist in the State of New Jersey.

Currently, the NJ Attorney Generals office has stopped JAR of Hope from selling raffle tickets online for a 2020 convertible Mustang that was donated by All American Ford in Old Bridge, NJ . This dealership has given us an incredible gift that is so generous and desperately needed at this time, 100% of the proceeds raised by ticket sales would be used to fund research for terminally ill children. Currently, 11-year-old Jamesy Raffone of Manalapan, 15-year-old Martin Cintron of Perth Amboy and dozens of other children living with Duchenne Muscular Dystrophy are suffering from this awful disease and need these tickets sold to fund an experimental drug treatment that just might save their lives. Please sign and share, we will do the rest.

PLEASE SIGN THIS PETITION!

JOIN US ON A “WALK FOR THEIR LIVES”

Tue, 24 Jan 2023 17:30:56 GMT

Joe Ippolito (our Treasurer) and I will be taking a 260-mile “walk” next month. A “Walk For Their Lives,” to be exact, from Washington, D.C. to Old Bridge, to raise awareness of Duchenne Muscular Dystrophy. Yes, you heard me right – from D.C. to NJ.

By now, you know that kids with Duchenne are in wheelchairs by their early-teens. Breathing through ventilators by their late-teens. And that there is no cure.

But we want the rest of the world to know, too. So Joe and I will be driving to Washington with our families, and they’ll drive back home while we “Walk For Their Lives.” We will start on Columbus Day, Oct. 12. We’ll be walking 33-35 miles a day and camping out each night.

On the night of Sunday, Oct. 18, we’ll be camping out at Sportika in Manalapan Township. And the next day, we will walk the last 26.2 miles (the length of a marathon). We will stop at the last sponsored checkpoint at All American Ford Old Bridge. Then it is on to the finish line at Lombardi Field in Old Bridge (near Old Bridge High School), where Mayor Owen Henry and Congressman Chris Smith will greet us.

And we would love for you to join us! Please feel free to join us anywhere along the route, and to leave anywhere (although you are invited to the Finish Line for the ceremonies!)

Our “Walk For Their Lives” will be sponsored by All American Ford in Old Bridge, and we’ll wind through Maryland and Pennsylvania before New Jersey. Our goal is to raise $50,000+ toward a cure for Duchenne Muscular Dystrophy…so that no parent will ever again have to hear the news that Karen and I heard seven years ago.

Please join us! It will be a joy to see our friends and neighbors as we head toward the finish!

For the latest updates, please follow our Facebook group, D.C. to All American Ford – Team Jamesy 24/7 .

To donate or to become a fundraiser, please visit the Donation Page .

-Jim Raffone

JAR OF HOPE FOUNDATION BRINGS SICK YOUNGSTER TO UNITED STATES FOR TREATMENT

Tue, 24 Jan 2023 17:28:43 GMT

It’s not easy to get a little Irish boy with a fatal illness to the United States for medical treatment, especially during COVID-19. But JAR of Hope has done it.

Alfie Pentony, 7, lives in Northern Ireland with his parents, Colleen and Jamie, and two brothers, according to a press release from JAR of Hope.

At the age of 4, Alfie was diagnosed with a very rare, always-fatal muscle-wasting disease called Duchenne Muscular Dystrophy. By their early teens, victims of Duchenne are in wheelchairs and by their early 20’s they are in graves after suffocating to death, according to the press release.

Alfie is participating in a year-long study called Operation Lifeline, conducted by the Salerno Center for Complementary Medicine in New York City, and sponsored by the JAR of Hope foundation of West Long Branch, which raises funds to research a cure for Duchenne.

Because of COVID-19, Alfie and his dad were initially denied entry into the United States for this month’s tests, according to the press release.

“This is a very promising study and we were determined to get Alfie here for the next phase, which is this week,” said Jim Raffone of JAR of Hope, who, with wife Karen, founded the organization in 2014 after their 4-year-old son James Anthony was diagnosed with Duchenne, according to the press release.

“So I began hitting the phone, to Congressman Chris Smith of New Jersey; Tom Lantos of Congress’ Human Rights Commission; Dr. John Salerno of the Salerno Center; and Patrick Smith, legal counsel to Congress’ Human Rights Commission, who was able to connect us to the U.S. State Department and the American Embassy in Ireland,” Raffone said.

In Operation Lifeline, four boys with Duchenne receive infusions of a chemical compound called JAR914 every three months and their muscle strength is measured before and after the treatment. The results have been very encouraging, according to the press release.

“We had brought Alfie here every three months for the treatments, since last October,” Alfie’s dad Jamie said. “But to get here now we needed assistance and JAR of Hope was really there for us. I don’t think we could have gotten Alfie here without them.”

“This is a ground-breaking study,” Raffone said. “For the first time, we are seeing results that may actually prolong the lives of kids with Duchenne … and maybe even lead to a cure. So when Alfie’s parents contacted us, we vowed we would not stop until we got him here.”

Finally, only weeks before this week’s phase of Operation Lifeline, Jamie Pentony and Alfie received permission to come to the United States and Alfie is undergoing the infusions right now, according to the press release.

WIN A 2020 FORD MUSTANG CONVERTIBLE

Tue, 24 Jan 2023 17:23:47 GMT

JAR of Hope and All American Ford are raffling off a brand new Mustang convertible to help fund a cure for Duchenne Muscular Dystrophy

JAR of Hope is a non-profit that raises money for research into a cure for Duchenne Muscular Dystrophy (DMD). They have a close relationship with the All American Auto Group , who will be donating a 2020 Ford Mustang Convertible to the cause. 100% of the proceeds will benefit JAR of Hope. You can purchase raffle tickets by visiting www.jarofhopemustang.com . DMD is a rare disease that causes muscles to degenerate, affecting about 20,000 children in the United States. It is a terminal children’s disease with no cure.

Jim Raffone is the founder and CEO of JAR of Hope. He founded the non-profit with his wife, Karen, after their son, Jamesy, was diagnosed with Duchenne Muscular Dystrophy. Doctors said that there was nothing they could do for Jamesy, but the Raffones refused to give up. They created JAR of Hope to raise awareness and funds for research into finding a cure for DMD. They are local to Central New Jersey.

“JAR of Hope is dedicated to directing funds to find a cure for Duchenne Muscular Dystrophy. No parent should have to hear from a doctor that nothing that can be done to help their child. Every raffle ticket that is purchased will help us get one step closer to a world with a cure,” said Jim Raffone, the founder and CEO of JAR of Hope. “I’d like to thank All American Ford for donating this beautiful Mustang to the cause. When I first saw it in their amazing showroom sporting our JAR of Hope green coloring, I knew we had to do something special with it and the Savino family was all for it.”

Raffle tickets for a chance to win this brand new 2020 Ford Mustang Premium Convertible are just $100 and 100% of the proceeds will benefit JAR of Hope. You can purchase your donation raffle tickets online at www.jarofhopemustang.com . They are limiting the amount of tickets to 1,000. The raffle will end on November 24th, 2020 and a winner will be announced. You can come check out the exact Mustang in this contest by visiting All American Ford in Old Bridge.

“ All American Ford has long been a proud partner of JAR of Hope. We are thrilled to be donating this Ford Mustang to help raise funds during a year in which fund raising was very hard to come by for charities.” says Jason Savino, Marketing Director for the All American Auto Group. “We encourage everyone in the community to check out the Ford Mustang on our showroom, tell your friends and family and share it like crazy on social media. Every little thing helps raise awareness and brings JAR of Hope closer to finding a cure for Duchenne Muscular Dystrophy.”

The Ford Mustang has been the top-selling sports car in the U.S. over the last 50 years. It is a truly iconic American name and the 2020 Ford Mustang lives up to it. The Mustang in this charity raffle is a 2020 Ford Mustang Premium trim with a special black accent package. It boasts a 2.3L EcoBoost engine, 10-speed selectshift automatic transmission and is a convertible. The exterior color is a beautiful Grabber Lime, a very similar tint to the JAR of Hope logo.

###

About the Mustang

VIN: 1FATP8UH7L5145086, Stock #: 200811, Model Code: P8U, Mileage: 50, MSRP: $41,865

About All American Auto Group

The All American Auto Group is one of the largest auto groups on the East Coast, owned by two partnering families – the Savino’s and the Selman’s. Family members are in the highest positions throughout the group and run the day-to-day operations. The auto group features 7 huge locations across New Jersey in Old Bridge , Point Pleasant , Brick , Hackensack and Paramus . They offer the largest selection, best prices and an award-winning experience throughout sales, service and parts. The All American Auto Group offers Ford, Subaru, Mazda, Isuzu Box Trucks, Jerr-Dan Tow Trucks and MOTOR TREND Certified Pre-Owned Vehicles. More information can be found at www.AllAmericanAutoGroup.com .

H360 HEALTHTALK: JIM RAFFONE | JAR OF HOPE

Tue, 24 Jan 2023 17:16:28 GMT

Dave is a business and technology strategist for the pharmaceutical and healthcare industry. With a heritage in healthcare marketing, communications, and technology, David has worked with pharmaceutical companies, the medical profession, research organizations and patient groups for over 20 years, completing various types of marketing and strategy projects across almost all disease areas.

David founded healtheo360 in 2013 after his sister-in-law was diagnosed with stage IV breast cancer and his mother with Alzheimer’s disease. After unsuccessful efforts with other support groups, due in part to the widening age gap between members of these groups, the family was forced to search for other outlets of social support. In response, healtheo360 was started. Visit them here.

RECAP OF THE RECORD-BREAKING 260-MILES FROM D.C. TO NJ

Tue, 24 Jan 2023 16:58:04 GMT

Manalapan Dad Walks Record-Breaking 260-Miles From D.C. To NJ For Son With Rare Disease

MANALAPAN, NJ – In just an 8 day period from Monday, Oct. 12 to Monday, Oct. 19, Manalapan resident Jim Raffone and a small team walked from Washington D.C. to Old Bridge, New Jersey. This record-breaking journey was all in an attempt to raise money for a cure for a fatal disease affecting his son.

Raffone’s 11-year-old Jamesy was born with Duchenne Muscular Dystrophy (DMD), a rare genetic disorder that affects some 20,000 boys in the U.S. It is one hundred percent fatal, and those with a diagnosis typically don’t live past their early twenties. Jamesy was diagnosed at age four and Jim recalls it being the worst day of his life.

In 2013, Raffone founded JAR of Hope, a nonprofit organization aimed at finding a cure for DMD. Within seven years, Jamesy’s parents have fundraised through galas and golf outings, push-up campaigns and even completed some of the hardest races on the planet, ranging from a seven-day 171-mile run through the desert to a 12-day trek throughout New Zealand and Australia.

With COVID-19 and the related closures, many of JAR of Hope’s planned fundraising events this year were cancelled. Jim knew he had to come up with something big to make up for lost time. The foundation desperately needed to continue to raise funds for a critical study involving numerous young boys with DMD.

JAR of Hope Treasurer Joe Ippolito and Jim came up with this idea for a record-breaking 260-mile trek. They started in Washington D.C. and made their way North, stopping at sponsored check points along the way to raise awareness. They were always met with an amazing response from the local communities. Their partnership with All American Ford in Old Bridge helped them connected with some Ford dealerships along the way – most got their local first responders involved and headed their own fundraising efforts in support.

The last sponsored checkpoint was All American Ford in Old Bridge, which is proudly family owned and operated by the Savinos and Selmans. The Savino family has long supported JAR of Hope as Jim Raffone was a customer and told his story to them some 5 years ago. Ever since, Jason Savino, Marketing Director for the All American Auto Group, and Jim Raffone have come up with some amazing fundraising ideas. Just earlier this year in January, they held the first ever charity poker event in the All American Ford in Old Bridge showroom which raised over $100,000 for JAR of Hope.

The Savino family presented a check to JAR of Hope for $88,000, which along with the dealership staff, they helped raised leading up to the event. After the stop at All American Ford in Old Bridge, it was off to the official journey finish line at Lombardi Field in Old Bridge. The All American staff joined Jim and the JAR of Hope family & team on the last 2 miles of the trek to the finish line ceremony. Old Bridge Mayor Owen Henry and Congressman Chris Smith were waiting for them at the field to present a JAR of Hope flag that was flown over the Capitol Building in D.C. as a sign of support.

Jim has called the journey one of the hardest in his life, mostly the fact of being away from his family for 8 days. It was a huge success though, raising much needed awareness across multiple states and getting much needed funding during such a tough year. JAR of Hope raised over $165,000! It captured the attention of the media and was covered by News12, ABC7 News, CentralJersey.com , Patch.co m , TheJournalsNJ.com, TheDad.com , New Jersey News Network and more!

From UFC.com – After finding self, Perez helps others in retirement

Tue, 26 Apr 2016 14:39:54 GMT

Article taken from UFC.com.

It’s about living your life how you want to live it.

Last August in Saskatoon, a 54-second knockout of veteran Sam Stout by a young and fairly unknown lightweight prospect named Frankie Perez culminated with an impromptu retirement inside the Octagon.

To everyone’s surprise, including his coaches and father, it was the 26-year-old from Howell, New Jersey – who had just won his first UFC bout – hanging up his four-ounce gloves.

It wasn’t an easy decision for Perez, but it was his call to make and to live with and, ultimately, he was happy with it.

“It was just something that felt right,” he said. “I never second guessed myself. I’ve been doing this since I was 17 years old. I never lived a normal lifestyle but I don’t regret the life I lived. I loved the fighting and the training, but I just came to grips with it that it was time to live my life the way I wanted to live it.”

Besides improving his record to 10-2, Perez took home a Performance of the Night bonus for less than a minute’s work. The outcome was a complete 180 from what he experienced seven months earlier in his UFC debut against Johnny Case. That January night in Boston, Perez felt like a shell of himself and, in the end, was finished for the first time in his pro career. The seeds of Perez’s eventual retirement on a high note were truly sewn from those low moments earlier in the year.

“Going into that first UFC fight, it was the worst of all situations,” Perez said. “I lost two family members back-to-back and I got TKO’ed. I hit rock bottom. Going into the camp against Sam, I thought there’s nothing that could happen to make it worse. It was a clean slate for me. There was nobody that could have been in front of me that day that was going to beat me.”

Win or lose against Stout, Perez was planning on walking away, as he told his mom, his brother, his two sisters and his best friend over the phone from his hotel room in Saskatoon after completing his final weight cut. Since then, Perez followed through on that promise to himself as he took a much needed break from the life of a full-time fighter. Instead of battling guys like Edson Barboza and former UFC lightweight champion Frankie Edgar in the gym six days a week, Perez traveled, had some fun, ate too much and got to spend time with his family.

While on this sabbatical of sorts, Perez came to peace with himself and the sport and remembered why he loved it. Recently, Perez has started training full-time again with the famed New Jersey team overseen by Mark Henry and Ricardo Almeida, and right now, his focus is helping others.

Professionally, Perez is Corey Anderson’s Muay Thai coach, was Barboza’s main sparring partner ahead of his UFC 197 win against Anthony Pettis and is teaching MMA classes at Nick Catone’s gym in Bricktown. Personally, Perez has become involved with a different fight – one against muscular dystrophy with Jim Raffone and his charity, JAR of Hope.

“Jim’s son was diagnosed with Duchenne muscular dystrophy, which is the rarest and most aggressive form,” Perez said. “And there’s no cure. Jim had been trying to get a hold of me to train him to fight. He really wants to do an exhibition fight to raise money for his son and this research that he’s investing in. We finally sat down and he laid everything out for me and I couldn’t say no. His son is basically dying in front of him and he’s trying anything he can to save him.”

Over the past year, Raffone and his seven-year-old son Jamesy have become celebrities in Monmouth County. Through JAR of Hope, Raffone, a father of three, has raised over $240,000 for research into the Duchenne muscular dystrophy that afflicts his son. Once a bodybuilder and personal trainer, he is doing everything he can think of to raise money to help combat his son’s degenerative disease and hopefully find a cure, from competing in push-up contests and running marathons to training for an amateur MMA fight.

Click here to learn more about JAR of Hope , or follow them on Twitter @JAR_of_Hope

"I’d like to thank the UFC for giving me the opportunity to put me on a bit of a pedestal to get my name out there and help organizations like (JAR of Hope) and bring some traffic their way." -- Frankie Perez

“He’s an incredible individual,” Perez said. “He’s not a young guy, but he is athletic. He runs these marathons like it’s nothing and goes back to work the next day. He doesn’t have any quit in him. I call him the ‘2016 John Q.’ He’s just an average guy who is willing to do anything to save his son. If helping him get ready for an amateur MMA fight is what he wants from me, then let’s do it.”

Their sights are set on an October debut for Raffone, and Perez has also enlisted fellow UFC veteran Dante Rivera – who is Raffone’s BJJ coach – to aid the cause.

“We’re giving Jim as much time as we can to raise as much money as we can to save his son’s life through JAR of Hope. I’d like to thank the UFC for giving me the opportunity to put me on a bit of a pedestal to get my name out there and help organizations like Jim’s and bring some traffic their way.”

Along with JAR of Hope, Perez is taking a cue from Raffone and he plans to raise money through his family’s Dead Serious MMA Promotion for multiple sclerosis research, as Perez's sister suffers from the disease. And on June 11, Perez will be lacing up a different type of gloves, as he will compete in a kickboxing bout as the main event for World Class Kickboxing Championships in North Bergen to raise money for JAR of Hope and the National Multiple Sclerosis Society.

In retirement, Frankie Perez's life is as physically demanding as ever. But with a renewed focus on helping others, he has the motivation to keep grinding.

jarofhope

Understanding the FDA’s New ELEVIDYS Warning: A Family-Centered Perspective

Agamree becomes 1st approved DMD treatment in Canada

Health Canada gives OK to therapy for patients 4 and older

Treatment designed to work with fewer side effects ﻿

Disability advocates let their voices be heard at symposium

People with Duchenne MD, other diseases have a place in shaping the future

Why Duchenne stories need global platforms ﻿

Representing the Duchenne community in public conversations ﻿

Exon-skipping therapy shows promise for DMD44

Del-zota helps ambulatory, nonambulatory patients, trial data show

On track for accelerated approval application ﻿

Trial shows treatment boosts dystrophin production ﻿

Latest News with Sareptas Drug for the Duchenne Community

Following second patient death, Duchenne muscular dystrophy families deserve answers about Elevidys

Understanding Aluminum Toxicity: What You Need to Know

What Is Aluminum?

How Does Aluminum Get Into Our Bodies?

Aluminum's Impact on the Brain and Neurodegeneration ﻿

Aluminum and Heavy Metal Toxicity: Inflammation and Systemic Effects

Testing for Aluminum Toxicity

Natural Approaches to Removing Aluminum from the Body

JAR of Hope Named 2025 Non-Profit of the Year by Monmouth Regional Chamber of Commerce

The Microbiome and Its Impact on Autism: Understanding the Gut-Brain Connection

Researchers Find 102 Genes Linked to Autism in One of the Largest Studies of Its Kind to Date

CBD improves behavior of kids with autism — with new studies testing children as young as 5

Children with autism are showing ‘dramatic improvement’ with this cheap drug — and doctor behind its research says it may one day prevent the disorder

Understanding Autism: A Biomedical Approach to Training and Treatment

The Genetic Component of Autism

The Role of Environment in Modifying Autism

The Mind-Body Connection

Conclusion

Sarepta's 27% Sell-Off Is 'Overblown,' 'Overdone' And 'Overly Bearish.' Here's Why It Happened.

'Very Unique' Case

Well-Known Side Effect

Transforming the Future of Duchenne Treatment: A Partnership for Progress

JAR of Hope is proud to share the groundbreaking partnership between Roche and Sarepta Therapeutics, aimed at reshaping the way Duchenne muscular dystrophy (DMD) is treated.

JAR of Hope Changes a Family's Life

A Familiar Name Joins the JOH Roster: Kimberly Buk

Kimberly Buk (formerly Kravitz) is a two-time Emmy Award-winning television journalist with a long string of notable accomplishments and awards. She’s a familiar face in the Tri-State area, in both print and electronic media. ﻿

Assistance for Families with Autism

Office of Autism

Breakthroughs in Stem Cell Research: How Mesenchymal Stem Cells (MSCs) Combat Duchenne Muscular Dystrophy

Understanding MSCs

MSCs in the Fight Against DMD

Clinical Trials and Promising Results

Challenges and Future Directions

Conclusion

Pfizer Update on Clinical Trial

'Time is against us': Half-marathon closing Route 9 to fund desperately needed drug trial

The Towncast – Jim shares his journey up Mt. Everset

JAR of Hope team climbs Mt. Everest to save the first child

Nebraska man joins fight against Duchenne muscular dystrophy

Franklin Square kickboxing marathon raises money toward Duchenne muscular dystrophy research

JAR of Hope presents National Pushup Campaign to all students

Will you accept this challenge Millstone Twp. & Upper Freehold Regional School Districts? ﻿

Italfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy

Hyperbaric Oxygen Treatment (HBOT) Study

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JOIN US ON A “WALK FOR THEIR LIVES”

JAR OF HOPE FOUNDATION BRINGS SICK YOUNGSTER TO UNITED STATES FOR TREATMENT

WIN A 2020 FORD MUSTANG CONVERTIBLE

JAR of Hope and All American Ford are raffling off a brand new Mustang convertible to help fund a cure for Duchenne Muscular Dystrophy

H360 HEALTHTALK: JIM RAFFONE | JAR OF HOPE

RECAP OF THE RECORD-BREAKING 260-MILES FROM D.C. TO NJ

Manalapan Dad Walks Record-Breaking 260-Miles From D.C. To NJ For Son With Rare Disease

From UFC.com – After finding self, Perez helps others in retirement

Treatment designed to work with fewer side effects

Why Duchenne stories need global platforms

Representing the Duchenne community in public conversations

On track for accelerated approval application

Trial shows treatment boosts dystrophin production

Aluminum's Impact on the Brain and Neurodegeneration

Kimberly Buk (formerly Kravitz) is a two-time Emmy Award-winning television journalist with a long string of notable accomplishments and awards. She’s a familiar face in the Tri-State area, in both print and electronic media.

Will you accept this challenge Millstone Twp. & Upper Freehold Regional School Districts?

Equal justice for Charities: allow Charities to sell raffles tickets on-line in NJ