STAT+: Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies 

Sarepta Therapeutics said that a young man died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy

Mar 18, 2025 - 14:45
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STAT+: Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies 

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 man 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 young man’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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