Patient dies following muscular dystrophy gene therapy, Sarepta reports

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the death Tuesday morning, sending the company’s shares plummeting in morning trading. Sarepta says the young man died of acute liver injury, a known side effect of the therapy. But the company says the severity of the patient’s case had not previously been seen. It’s the first known patient death with the therapy, which has been used in more than 800 patients. The therapy was approved in 2023 despite concerns from some Food and Drug Administration’s scientists about its effectiveness.

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