The FDA is investigating the June 7 death of an 8-year-old Brazilian boy receiving Sarepta’s Duchenne muscular dystrophy gene therapy Elevidys amid voluntary shipment suspensions. Sarepta and Roche, holding rights outside the U.S., maintain the fatality is unrelated to treatment per Brazilian authorities. This development follows recent patient deaths and an EMA recommendation against Elevidys approval in Europe for young ambulatory patients. The safety review impacts global availability in FDA-approved markets and underscores the scrutiny on emerging gene therapies' risk profiles.