Roche announced new dosing restrictions for Elevidys, a gene therapy for Duchenne muscular dystrophy (DMD), due to two fatal cases of acute liver failure in non-ambulatory patients. Treatment for non-ambulatory patients is immediately paused in clinical trials and discontinued commercially. The benefit-risk profile remains positive for ambulatory DMD patients. Health authorities and physicians are being informed of the changes to ensure appropriate patient care. The decision follows a reassessment of the benefit-risk ratio for non-ambulatory patients. Roche expresses deep sadness regarding the loss of the two patients and prioritizes patient safety. Several ongoing clinical trials involving Elevidys are temporarily halted or paused. Elevidys is approved in eight territories for DMD treatment. Roche and Sarepta Therapeutics are collaborating on Elevidys development and commercialization. The ongoing clinical trials for ambulatory patients will continue as planned.