Sarepta Therapeutics faces major scrutiny following the deaths of three patients linked to its gene therapies for muscular dystrophies. Two teenage patients treated with Elevidys for Duchenne muscular dystrophy died from acute liver failure, prompting Sarepta to pause shipments for non-ambulatory patients and the FDA to investigate. A third death involved a 51-year-old man in a trial for limb-girdle muscular dystrophy, also due to liver failure. The FDA has requested that Sarepta halt all shipments of Elevidys, and is considering withdrawing it from the market. Sarepta has agreed to add a black box warning for acute liver injury to Elevidys and is exploring prophylactic immunosuppression strategies. The controversies have led to significant organizational changes at Sarepta, including a 36% workforce reduction aimed at securing financial stability and refocusing the company’s pipeline.