Sarepta Therapeutics has faced mounting safety concerns after three patients died from acute liver failure linked to its gene therapies in 2025. The most recent death involved a patient in an early-stage trial for limb-girdle muscular dystrophy (LGMD), adding to two prior fatalities associated with Elevidys, Sarepta's gene therapy for Duchenne muscular dystrophy (DMD). In response to these events, the U.S. Food and Drug Administration (FDA) requested Sarepta to voluntarily halt shipments of Elevidys and placed clinical trials for multiple gene therapy products on hold. Sarepta initially refused the FDA's request but later reversed course, announcing a voluntary pause on all Elevidys shipments in the U.S. to address safety labeling and regulatory concerns. The FDA also revoked the platform therapy designation for the AAVrh74 serotype used in these treatments and restricted Elevidys use to ambulatory DMD patients. Following the pause, Roche, Sarepta's partner, suspended Elevidys shipments in some countries outside the U.S. The FDA investigated the death of an 8-year-old boy in Brazil who had received Elevidys; Brazilian authorities and Roche deemed the death unrelated to the therapy. After completing its review, the FDA recommended lifting the voluntary hold on Elevidys shipments for ambulatory patients, allowing Sarepta to resume distribution for this group while the hold remains for non-ambulatory patients. This regulatory development led to a significant rebound in Sarepta's stock price and upgrades from several analysts. Despite the FDA's clearance for ambulatory patients, a senior FDA official described Sarepta's path to fully restoring Elevidys to the market as "arduous and treacherous."