By I. Edwards HealthDay Reporter<\/b>
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WEDNESDAY, March 19, 2025 (HealthDay News) — A patient has died while receiving Elevidys<\/a>, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment.<\/p>\n The company behind the drug, Sarepta Therapeutics<\/a>, said that the patient suffered acute liver injury, a known side effect, The Associated Press <\/em>reported. However, the “severity” of this case had not been seen before, the company noted.<\/p>\n Sarepta did not release details about the patient but said a recent infection may have played a role in the liver injury.<\/p>\n The company plans to update the prescribing information for Elevidys to reflect this case.<\/p>\n Shares of Sarepta, based in Cambridge, Massachusetts, dropped more than 27% on Tuesday, closing at about $73 per share.<\/p>\n Elevidys, which costs $3.2 million for a one-time infusion, was the first gene therapy approved<\/a> in the U.S. for Duchenne muscular dystrophy, a rare condition that causes muscle weakness, mobility loss and early death in males.<\/p>\n The FDA first granted the drug expedited approval in 2023, despite some scientists questioning its effectiveness.<\/p>\n In 2024, the agency expanded full approval to include patients 4 and older, regardless of mobility status.<\/p>\n The therapy works by using a disabled virus to insert a gene that helps the body produce dystrophin, a protein crucial for muscle function. Sarepta said more than 800 patients have received Elevidys so far, The Associated Press <\/em>reported.<\/p>\n Sarepta has secured FDA accelerated approval for three other Duchenne drugs since 2016. But none have yet been confirmed to be effective and further studies are still ongoing.<\/p>\n More information<\/strong><\/p>\n Learn more about Elevidys<\/a>.<\/p>\n