Newly diagnosed? Read the latest insights on what to expect next.
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2026 Muscular Dystrophy Association conference highlights recent advancements
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A snapshot of William’s life with Duchenne muscular dystrophy
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Gene therapy GNT0004 shows lasting benefit for boys with Duchenne
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How to ‘Light Up for Rare’ on Rare Disease Day
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What Rare Disease Day means to me
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PTC Therapeutics withdraws ataluren application after FDA review