FDA expands approval of Duchenne muscular dystrophy gene therapy Elevidys to include 4+ boys and men with DMD gene mutation.
The FDA has expanded its approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD), to include boys and men with a confirmed mutation in the DMD gene who are at least 4 years of age. Developed by Sarepta Therapeutics, the treatment targets the genetic root cause of DMD and has shown functional benefits in both ambulatory and non-ambulatory patients. The expanded approval offers renewed hope and progress in the fight against DMD.
June 20, 2024
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