Sarepta Therapeutics has suspended shipments of its Duchenne muscular dystrophy gene therapy, Elevidys, specifically for non-ambulatory patients following a second fatal case of acute liver failure. Both deaths occurred in boys with advanced disease stages who had lost the ability to walk. In response, Sarepta is convening an independent expert panel to evaluate an enhanced immunosuppressive regimen aimed at reducing liver toxicity, with discussions ongoing with the FDA. Despite these events, no changes have been made for ambulatory patients, and routine corticosteroid administration continues. The safety concerns have caused significant stock price declines, raising questions about the gene therapy's future and regulatory outlook.