FDA expands approval for Duchenne muscular dystrophy gene therapy

The U.S. Food and Drug Administration has expanded the approval of Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in individuals ?4 years with DMD with a confirmed mutation in the DMD gene. Elevidys is a single-dose, intravenous recombinant gene therapy designed to produce Elevidys micro-dystrophin, a shortened protein (138 kDa, versus the 427 kDa dystrophin protein of normal muscle cells) that contains selected domains of the dystrophin protein present in normal muscle cells. The FDA granted traditional approval for ambulatory individuals Read More

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