gene therapy, muscular dystrophy
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A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys.
Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other parents still push for the therapy.
Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and clinical trial pause.
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today provided a safety update regarding ELEVIDYS (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy,
Sarepta's stock drops on ELEVIDYS safety concerns, yet potential remains for ambulatory patients. Click here to read an analysis of SRPT stock now.
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