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The neuromuscular disease community received encouraging news this week as Edgewise Therapeutics announced the sale of sevasemten and its Becker and Duchenne muscular dystrophy programs to Servier, a global pharmaceutical company with a strong track record of developing and delivering innovative therapies to patients worldwide.  CureDuchenne invested in Edgewise in 2019, recognizing the potential of their approach. This acquisition is…

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It’s no secret that MDA advocates are a force on Capitol Hill and have routinely commanded the hall of Congress during MDA on the Hill over the years. Jennifer Lane and Rep Aderholt But what if MDA teamed up with volunteers and advocates from other organizations to come together as one powerful group? That is…

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Morimoto–Ryu–Malicdan neuromuscular syndrome (MRMNS) is an inherited condition that was first reported on in late 2024. It is caused by variants in the RFC4 gene and is classified as a congenital myopathy, primarily affecting skeletal muscles and in some cases the nervous system. Much about the symptoms and progression of this newly discovered condition remains…

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Here are some facts about me: I am an introvert, I am quiet, and my strongest voice is the written word. I grew up in the foster care system and experienced childhood trauma that makes it hard for me to know how to be around people. I am a mom to seven children: Lexi, 25,…

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Researchers at Vor Bio are conducting a global phase 3 clinical study (UPSTREAM MG) for adults with generalized myasthenia gravis (gMG) who are AChR or MuSK+. The study will evaluate whether treatment with the investigational drug telitacicept can reduce the level of disease-causing B cells and autoantibodies, and improve muscle weakness and fatigue, in people…

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No treatments are currently approved and available for limb-girdle muscular dystrophy type 2i (LGMD2i) in the U.S., but that may change later this year. The U.S. Food and Drug Administration (FDA) has accepted an application from Bridgebio Pharma seeking approval for its experimental oral therapy BBP-418. The agency granted the application priority review, reducing the…

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The MDA Quest Media Product Guide is designed to help you find the products you need to live a more independent, stylish, fun, and all-around great life. All the products you see here were chosen by MDA Ambassadors, who shared exactly how each product helps them in their daily lives. Find something for yourself, or…

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During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, a friend of mine shared a poem by May Sarton that prompted the question: “Do I think of my body as an old friend or as an enemy?” I realized that my attitude toward my body has grown worse with recent difficulties. I…

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As someone living with spinal muscular atrophy (SMA), I know how important it is for children with disabilities to have full access to education. With accommodations for my mobility, I took honors and AP classes in high school, then went on to earn a master’s degree at Emerson College. I’m using my education to pursue…

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Neuromuscular diseases can impact muscles throughout the body, including the heart. Because heart health is essential to overall health, researchers are working to understand how different neuromuscular diseases affect the heart and why effects can vary between people with the same diagnosis. There are still open questions, but over the last decade, scientists have learned…

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