STAT+: Patient 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 - 14:05
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STAT+: Patient who received Elevidys, Sarepta’s Duchenne gene therapy, dies 

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

It is the first known death linked to the treatment. He 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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