Patient dies in Sarepta gene therapy trial, adding to safety concerns

A 51-year-old man died last month after receiving an experimental gene therapy developed by Sarepta Therapeutics for an ultra-rare form of muscular dystrophy, a company spokesperson confirmed Thursday. 

He is the third patient to die after getting a Sarepta gene therapy this year, all tied to liver issues. 

Two teenage boys died after receiving Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy, forcing the company to take the drug off the market for older patients. The 51-year-old man was in a clinical trial for limb girdle muscular dystrophy, a collection of rarer and generally slower-moving conditions.

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