A US-based gene therapy, Elevidys, intended to treat Duchenne muscular dystrophy, has been halted by regulators due to safety concerns. The FDA’s request for Sarepta Therapeutics to stop shipments of the treatment led to the company’s decision. As a result, thousands of patients who were awaiting the treatment have had their appointments postponed indefinitely.
The news has caused widespread confusion and heartbreak among families affected by Duchenne muscular dystrophy, with many fearing the loss of a potentially life-changing treatment. In an email, Stephen Chrzanowski, co-director of the Duchenne Program at UMASS Chan Medical School, emphasized that “kids’ lives and families’ well-beings are at stake” in this decision.
The company had initially refused the FDA’s request to halt shipments, but reversed course after receiving the agency’s final guidance. The future of Elevidys now hangs in the balance, with a senior FDA official describing its path back to market as “arduous” and “treacherous”.
Source: https://www.statnews.com/2025/07/24/duchenne-muscular-dystrophy-families-sarepta-elevydis