To benefit the Muscular Dystrophy Association
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Dear Friends,
This will be the 49th year our family has worked with an amazing team of volunteers and supporters to raise money for the Muscular Dystrophy Association. Please consider joining our "Telethon Team" to help support research, treatments, and find cures for muscle disease. Your help is their hope!
Telethon Party Schedule
Hingham Community Center
70 South Street
Hingham, MA. 02043
Sunday August 31st – (11am - 4pm)
Fun – Food - Entertainment
· Live Entertainment
· Bouncy Houses
· Pony Rides
· Games
· Balloon Art
· Cookout
· Raffles
· Silent Auction
· T-Shirts
· Awards
Tolman MDA Telethon Party
MDA Reflects on 75 Years of Research and the Precarious Times Ahead
Key Takeaways
- MDA's 75-year progress includes gene discoveries and drug approvals for DMD and SMA, emphasizing the importance of sustained research funding.
- Advocacy efforts target Medicaid coverage, newborn screening expansion, and legislative reauthorizations to prevent loss of access to care and services.
- Federal policy uncertainties pose risks to neuromuscular disease advancements, with Medicaid cuts threatening access for 45% of affected individuals.
- The establishment of a government-funded national registry for neuromuscular diseases is proposed to support broader gene therapy coverage.
Speakers from the Muscular Dystrophy Association (MDA) highlighted 75 years of progress in understanding and treating neuromuscular diseases but also raised concerns about how potential funding cuts could impact this community.
Neuromuscular disease researchers, clinicians, and advocates gathered to celebrate 75 years of progress at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference in Dallas, Texas, but they also noted that these gains could be reversed if cuts to Medicaid and research funding proceed.
Before the keynote address by former FDA Commissioner Robert Califf, MD,1 the MDA Conference attendees heard from Sharon Hesterlee, PhD, chief research officer at MDA, and Paul Melmeyer, MPP, executive vice president of public policy and advocacy at MDA. Hesterlee recapped the research strides that have been made in the 75 years since MDA was founded—which were also displayed in the windows of the hallway leading to the ballroom—and Melmeyer followed with a look at the year ahead in the association’s public policy and advocacy efforts.
Research strides were displayed in the windows of the hallway leading to the ballroom. | Image Credit: Christina Mattina
75 Years of Research Progress
According to Hesterlee, when MDA was in its infancy in the 1950s, there was some awareness of muscular dystrophy as a set of diseases that cause muscle weakness and run in families, but there was little understanding of the underlying causes. By 1986, MDA-funded research had discovered the dystrophin gene that causes Duchenne muscular dystrophy (DMD), but as the largest gene in the genome, it was too large to fit in an adeno-associated virus vector, and the first gene therapy trial in DMD wouldn’t begin until 2006.
Meanwhile, trials commenced for gene therapies to treat limb girdle muscular dystrophy in 1999, and nusinersen was approved for spinal muscular atrophy (SMA) in 2016, which Hesterlee described as “the point at which we realized that this disease that was completely intractable—that was absolutely terrible, that was the leading genetic cause of death in infants—was tractable, that you could actually change the expression of the gene and get a good outcome.” Now, there are 4 approved drugs for DMD, and tofersen has been approved to treat amyotrophic lateral sclerosis.
It's not just drugs that MDA is active in funding: Hesterlee also mentioned the creation of the MOVR Data Hub in 2013, which aims to aggregate longitudinal clinical data across neuromuscular diseases, and the Kickstart Program, which will encourage research in ultrarare disease states.
“We now have 28 drugs approved in neuromuscular disease, and building this entire ecosystem is so important to maintain,” Hesterlee said. “The point is research funding works. You see the pipeline, you see where these things come from, and that’s in danger with the cuts that have been impacting the NIH,” referring to cuts to National Institutes of Health funding that have been temporarily blocked by a federal judge.2 “We’re going to lose a generation of scientists right now if we can’t step in and help,” she warned.
A Busy Year Ahead for Advocacy
Melmeyer’s talk also covered some significant strides but warned of progress that could be lost without effective advocacy. For instance, in the past year, the neuromuscular disease community pulled together to achieve accessible air travel reforms, updates to disability rights legislation, and 100% of states now screening newborns for SMA. Looking at the year ahead, the MDA is focused on bills to reauthorize Pediatric Rare Disease Priority Review Voucher program, streamline Medicaid coverage across state lines, cover genetic counselors in Medicare, and expand newborn screening for Pompe disease and DMD.
However, there are significant concerns on the horizon, including lawsuits invalidating progress in disability protections, dismantling of federal offices such as the Department of Education, and threats to disability inclusion efforts, said Melmeyer. Proposed cuts to Medicaid are of particular concern,3 given that 45% of those with neuromuscular diseases are covered by Medicaid.
“If [the] federal government were to drastically cut the amount of funding that they give to states to cover the Medicaid programs, we know that our community will lose access to care,” Melmeyer said. “We know that they’ll lose access to services. We know that they’ll lose access to the clinics, the treatments, to community-based services, and more.” He called on attendees to visit MDA’s Medicaid advocacy page and make their voices heard.
Federal Policy on Speakers’ Minds
The unpredictable federal policy landscape was brought up several times throughout the MDA Conference sessions. In a session about securing gene therapy coverage,4 Chamindra Laverty, MD, neurologist at UC San Diego Health, said that a government-funded national registry for neuromuscular diseases is the ideal solution to collate the data that payers will require for broader coverage—but it’s a solution that is likely not forthcoming: “I’m not sure how valid it’s going to be moving forward, but that is the only way that I can think of, because otherwise it’s difficult for a foundation or a nonprofit year after year to fund such a thing.”
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