Sarepta's Elevidys Gene Therapy Falls Short in Pivotal Trial

Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy failed to improve muscle function compared to a placebo in a pivotal clinical trial, raising doubts about FDA approval and disappointing patients and doctors eagerly awaiting the treatment. Although all patients in the study showed improvement and secondary measurements indicated the drug's effectiveness, the primary endpoint was not met. Trading in Sarepta shares was halted following the release of the study results.