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The adeno-associated virus (AAV) vector-based gene therapy delandistrogene moxeparvovec-rokl (Elevidys – Sarepta) received accelerated approval from the FDA in 2023 for treatment of ambulatory children 4-5 years old with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene.1 It has now received full approval for use in ambulatory patients ≥4 years old and accelerated approval for use in nonambulatory patients with DMD.
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A 2024 study studied this question.
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