The FDA is investigating the death of an 8-year-old Brazilian boy treated with Sarepta Therapeutics’ Duchenne muscular dystrophy gene therapy Elevidys, following previous patient fatalities linked to the drug. Shipments have been voluntarily suspended in the US and other countries. Sarepta maintains that the death was unrelated to the therapy, with Roche, which holds rights outside the US, echoing this assessment. Meanwhile, the European Medicines Agency recommended against Elevidys approval in Europe for young ambulatory patients. These events underscore ongoing safety concerns with gene therapies for neuromuscular disorders.