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The FDA approved delandistrogene moxeparvovec-rokl (Elevidys, Sarepta Therapeutics) in June to treat Duchenne muscular dystrophy in ambulatory pediatric patients aged 4 through 5 who have a confirmed DMD gene mutation.


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,Methods Clinical Development ,National Organization For Rare Disorders ,Sarepta Therapeutics ,Molecular Therapy ,Image Credit ,Ther Methods Clin ,Updated June ,Updated August ,Delandistrogene Moxeparvovec ,Elevidys ,Dmd Gene Mutation ,

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