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Life with Lily: The Right to Access My Education Fully
“Lily, I think you’re old enough now to advocate for yourself in your 504 meeting today.” I remember my mom saying that like it was a small thing. It wasn’t. I was in middle school, nervous, unsure, and still coming to terms with the idea that having a disability meant I’d have to explain my…
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I’m waving goodbye to acting, but I’m not about to exit the stage
When I was 13 and fresh from spinal fusion surgery, I sat in a West End theater in London with my family, watching “Les Misérables.” The music stirred my soul and, for a fleeting moment, I imagined myself on stage. But that dream quickly faded. Accessibility barriers at school here in Singapore stopped me from…
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Edgewise Therapeutics: Focused on Muscle Disease
Developing a novel therapeutic designed to protect muscle in Becker and Duchenne muscular dystrophy Watch HERE The post Edgewise Therapeutics: Focused on Muscle Disease appeared first on CureDuchenne.
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Your Legacy Can Help Cure Duchenne
Legacy Giving August is Make-a-Will Month — the perfect time to reflect on how you can protect what matters most: your loved ones and the causes that hold a special place in your heart through legacy giving. At CureDuchenne, we know that a cure is within reach — and we’re accelerating that future every day…
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Early data for experimental DM1 therapy SRP-1003 coming soon
Preliminary data are expected this year for a Phase 1/2 clinical trial testing the investigational therapy SRP-1003 in people with myotonic dystrophy type 1 (DM1), according to an announcement from Sarepta Therapeutics. The trial recently hit one of two prespecified enrollment targets, prompting a review of safety data. The Phase 1/2 study (NCT06138743) is being…
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Community Members Reflect on MDA’s Impact
From our earliest days, MDA’s mission has been centered on ensuring that individuals and families living with neuromuscular diseases have the resources, care, and support they need to live stronger and more independently. Our community’s resilience and strength have propelled us to continually improve and find new ways to offer hope, connection, and community. We…
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MDA Community Programs Support and Empower
For 75 years, MDA has been dedicated to standing by your side, offering hope, connection, and community. From the earliest days, our mission has been centered around ensuring that you have the resources, care, and support you need to live stronger and more independently. How we accomplish our mission has evolved through the years —…
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Progress Now: Research News and Updates Across MDA Diseases Quest Magazine Issue 3, 2025
New approvals New Treatment Approved for gMG In April, the US Food and Drug Administration (FDA) approved Johnson & Johnson’s nipocalimab-aahu (IMAAVY) for the treatment of people ages 12 and older living with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody-positive. This is a significant advancement for the…
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Neuromuscular Disease Research: Going Strong
Is hope foolish? For 75 years, MDA has been answering with a resounding, “No.” When New York businessman Paul Cohen founded MDA in 1950, very little was known about neuromuscular disorders, which, at the time, were usually referred to simply as muscle disease. Only one person was interested in studying these diseases: Ade T. Milhorat,…
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MDA Summer Camp Magic
In 1955, MDA held its first Summer Camp in Sussex, New Jersey. Sixteen campers from New York boarded a bus and headed to a rustic yet accessible campsite, where they explored and built camaraderie, free from the typical physical and societal limitations. One of the first to be led by a voluntary health organization, this…
