TOGETHER WE CAN FIND A CURE

Let's Save the First Child from Duchenne muscular dystrophy

TOGETHER WE CAN FIND A CURE

Let's Save the First Child from Duchenne muscular dystrophy

Make a difference that lasts a lifetime!

Your donation of only 13¢ a day ($4/month) will help fund our clinical trial.

Make a difference that lasts a lifetime!

Your donation of only 13¢ a day ($4/month) will help fund our clinical trial.

PENNIES FOR HOPE

13 cents a day can save a child

Make a difference that lasts a lifetime!

Your donation of only 13¢ a day ($4/month) will help fund our clinical trial.

Make a difference that lasts a lifetime!

Your donation of only 13¢ a day ($4/month) will help fund our clinical trial.

TOGETHER WE CAN FIND A CURE

Let's Save the First Child from Duchenne muscular dystrophy

Make a difference that lasts a lifetime!

Your donation of only 13¢ a day ($4/month) will help fund our clinical trial.

Make a difference that lasts a lifetime!

Your donation of only 13¢ a day ($4/month) will help fund our clinical trial.

JAR of Hope is on a mission to set a world record by assembling the largest group to perform 10 squats simultaneously—all in support of children battling Duchenne Muscular Dystrophy.


Why? Because children with Duchenne typically lose the ability to walk by age 12 and will never be able to rise from a chair again. This event is a powerful way to stand—literally and figuratively—with those who no longer can.

GET YOUR TICKETS!

Staten Island Ferry Hawks
May 4, 2025 – 1:30pm
Jersey Shore Blueclaws
May 22, 2025 – 6:35pm
Miami Marlins
June 22, 2025 – 1:40pm
GET YOUR TICKETS

Because no child should have to suffer like this.

Duchenne muscular dystrophy (DMD) is one of the rarest diseases on Earth. DMD is a fatal, muscle-wasting disease you are born with. It occurs once in every 3,500 live births and effects mostly boys. In the United States, with a population of over 330,000,000 people, only about 20,000 children are stricken with Duchenne. And there is no cure.


Our goal is to find a cure for Duchenne muscular dystrophy. With organized fundraising events and support from you, we can continue our mission and turn the dream of finding a cure into a reality.

LEARN MORE

Together, JAR of Hope has

RAISED $7 MILLION+

HELPED OVER 25+ FAMILIES

SPREAD AWARENESS GLOBALLY

Together, JAR of Hope has

RAISED $7 MILLION+

HELPED OVER 25+ FAMILIES

SPREAD AWARENESS GLOBALLY

Duchenne Muscular Dystrophy Clinical Trial 

We have partnered with the University of Florida College of Medicine and RESTEM LLC to to initiate an important trial for Duchenne muscular dystrophy (DMD).


Authorization has been granted to begin Phase 1 of the trial. Here, a "randomized, double-blind, placebo-controlled Cross Over study to assess the safety, tolerability, and preliminary efficacy of Umbilical Cord Lining stem cells (ULSC) in Pediatric patients with Duchenne muscular dystrophy (DMD).”

LEARN MORE ABOUT THE TRIAL

In the News

March 19, 2025
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. 
December 10, 2024
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.
August 6, 2024
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.” 
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