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Gene Therapy for Duchenne Muscular Dystrophy Approved by FDA

FDA announced on June 22, 2023 the approval of Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of children ages four through five years that have Duchenne muscular dystrophy (DMD) and do not have a pre-existing medical reason that would prevent this kind of treatment. DMD is a rare genetic condition that leads to weakness and “wasting away” of the body’s muscles due to a defective gene, which creates an absence of a protein that keeps muscles intact.

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