Sarepta Therapeutics’ gene therapy Elevidys will be available to Duchenne muscular dystrophy (DMD) patients who are at least 4 years old, regardless of whether they can walk, the FDA said Thursday.
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Pfizer’s investigational Duchenne muscular dystrophy gene therapy fordadistrogene movaparvovec has been hit with another patient death, forcing the pharma to pause dosing in its Phase III study.