A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys.

Sarepta suspends Elevidys use in non-ambulatory Duchenne patients after two liver failure deaths, triggering FDA review and clinical trial pause.

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 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,