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Sarepta Therapeutics reports a patient death due to liver injury while using its muscular dystrophy gene therapy Elevidys.
Sarepta Therapeutics reported a patient death while using its gene therapy Elevidys for muscular dystrophy.
The patient, a young man, died from acute liver injury, a known side effect, marking the first fatality linked to the therapy.
Elevidys, which was expedited for approval in 2023 despite concerns about its effectiveness, has been used in over 800 patients.
Sarepta plans to update the therapy's prescribing information, and its shares fell by over 23%.
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Sarepta Therapeutics informa de la muerte de un paciente debido a una lesión hepática durante el uso de su terapia genética de distrofia muscular Elevidys.