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Annual campaign raises $641K to send youths to MDA Summer Camp
This year’s Be Their Muscle campaign has raised more than $641,000 to support MDA Summer Camp for children and adolescents with muscular dystrophy and other neuromuscular diseases, and to help advance research and care for these patient populations. Resulting from a partnership between the Muscular Dystrophy Association (MDA) and Burn Boot Camp, this year’s campaign has included 400…
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Preparing for the Deramiocel FDA Advisory Committee Meeting: How to Submit Written Testimony
The FDA has scheduled an Advisory Committee meeting for July 29, 2026 to review Deramiocel, Capricor’s investigational cell therapy for Duchenne muscular dystrophy. The meeting will be held virtually, and a live stream will be available for public viewing. The Duchenne community is actively working together to prepare for this important meeting. Following the announcement,…
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MDA Ambassador Guest Blog: The Part I Let Belong: Learning to Embrace My Disability Identity
John Scurto is a 29-year-old MDA Ambassador from Boca Raton, Florida, who lives with spinal muscular atrophy (SMA) and is a power wheelchair user. He holds an MBA and a graduate certificate in Disability Studies, and he is especially interested in the intersection of business and disability inclusion. With both lived experience and academic training,…
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Clinical Research Alert: Clinical Study in Children with SMA
Researchers at Teachers College, Columbia University (New York) are seeking children living with spinal muscular atrophy (SMA) who are receiving disease-modifying therapy to participate in a pilot study exploring the integration of targeted rehabilitation strategies. This intervention aims to strengthen muscles, enhance neuromuscular coordination, and reduce fatigue, ultimately supporting improved movement and functional abilities in children with…
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Highlighting the lived experiences of MD patients at conferences
I have been involved with the annual Parent Project Muscular Dystrophy (PPMD) conference since 2002. Back then, the conference was attended by fewer than 50 people, almost no one living with muscular dystrophy (MD), and no representatives from pharmaceutical companies. The sessions were presented by scientists who did not speak in layman’s terms. As someone…
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Scientists discover why exercise reverses muscle aging
Researchers have uncovered a molecular “switch” that helps explain why exercise keeps ageing muscles healthy. By reducing levels of a gene called DEAF1, physical activity allows older muscles to clear out damage, repair themselves, and maintain strength.
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I now appreciate it when someone says, ‘You have your hands full’
“You have your hands full.” I have heard that said hundreds of times over the past 25 years, and I hated hearing it. I always had a response ready: “Sure, but my heart is fuller,” I would say. However, I’m currently in a season of life and caregiving where I appreciate that someone notices that…
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New calculator reveals whether you should really worry about statin side effects
Scientists at the University of Oxford have created a calculator that predicts a person’s individual risk of serious muscle disorders from statin medications. Their analysis found that more than 98% of people who qualify for statins are at low risk for these rare complications, despite widespread concerns about side effects. The study also revealed that…
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MDA’s Guide to the Rehabilitation Act of 1973
The Rehabilitation Act of 1973 (Rehab Act) laid the foundation for disability civil rights in the United States, serving as the first major federal law to prohibit disability-based and establish accessibility requirements in federally funded programs and services. Joel Cartner For people living with neuromuscular diseases and other disabilities, the Rehab Act helped transform access…
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FDA review begins for full approval bids on 2 Duchenne therapies
The U.S. Food and Drug Administration (FDA) is expected to decide by Feb. 28, 2027, whether to grant traditional approval to Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). Both medications are currently approved in the U.S. under the FDA’s accelerated approval pathway,…
