Sarepta Therapeutics said Tuesday that a 16-year-old boy died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy, the first known death linked to the treatment.
The teenager suffered liver failure, a side effect that has been seen with other gene therapy treatments over the last two decades.
Sarepta said in its statement that a recent cytomegalovirus infection could have also contributed to the patient’s death, putting him at heightened risk. The company noted that over 800 patients have now been treated with the therapy.
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