Report: Failed Trials, Yet Full FDA Approval of a Duchenne Muscular Dystrophy Gene Therapy
View report
View press release
In separate regulatory decisions in 2023 and 2024, the Food and Drug Administration (FDA) approved a novel gene therapy for the rare but deadly childhood-onset disease Duchenne muscular dystrophy. This report describes the two ill-advised approvals, explains why they are examples of actions FDA leaders should avoid in the future, and makes recommendations for how the FDA should change the way it resolves controversial regulatory decisions.