Sarepta Therapeutics presented new safety data for its Duchenne Muscular Dystrophy gene therapy Elevidys, showing zero ambulatory patient deaths from acute liver failure among over 1,000 treated individuals. However, a notable portion experienced acute liver injury, with a small subset requiring hospitalization. These findings come amid regulatory scrutiny following fatalities in non-ambulatory patients, prompting safety warnings and treatment pauses for certain populations. Sarepta continues risk mitigation efforts as it advances clinical use of this promising gene therapy.