Sarepta reports second death case in its gene therapy

date
16/06/2025
Sarepta Therapeutics announced that another patient who received its gene therapy for a rare muscle disease has died from acute liver failure. The company stated that this death occurred in a patient receiving the one-time treatment drug Elevidys, and three months ago, a teenager died after receiving the treatment. Both cases occurred in patients with Duchenne muscular dystrophy who are unable to walk. The company has paused a clinical trial, stopped shipping the drug to non-ambulatory patients, and is considering strengthening immunosuppressive regimens for these patients.