Data on Delandistrogene Moxeparvovec Can Inform Future Gene

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 ,