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Why This Study Matters Now
Duchenne muscular dystrophy, or DMD, is one of the most devastating inherited childhood muscle diseases. Caused by mutations in the dystrophin gene, it leads to progressive muscle weakness, loss of walking ability, heart and lung complications, and shortened lifespan. For years, corticosteroids such as prednisone and deflazacort have been a cornerstone of treatment because they can slow functional decline. But that benefit comes at a price: weight gain, slowed growth, bone problems, behavioral side effects, and other toxicities that many families know all too well.
