Sarepta Therapeutics reported that a confirmatory study for two of its exon‑skipping therapies in Duchenne muscular dystrophy did not meet its primary endpoint. Despite the negative readout, the company said it will request full FDA approval based on the totality of evidence and prior accelerated‑pathway interactions. The move sets up a regulatory debate over the sufficiency of existing data and the agency’s standards for confirmatory trials in rare neuromuscular diseases.