The FDA has resumed clearance for Sarepta Therapeutics' Elevidys gene therapy targeting ambulatory patients with Duchenne muscular dystrophy after a thorough safety review. This follows a temporary halt prompted by concerns related to patient deaths, including an 8-year-old Brazilian boy whose death was ultimately deemed unrelated to treatment. While shipments to younger, ambulatory patients have restarted, the FDA maintains a hold on non-ambulatory patients as investigations continue. Sarepta and Roche, its ex-US rights holder, remain committed to transparency and patient safety amid regulatory scrutiny.