FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook.

Although the trial concluded in November 2025, it ultimately failed to meet its primary clinical endpoint; Sarepta Therapeutics announced top-line results, stating that the change from baseline in the 4-Step ascend velocity at week 96 did not reach statistical significance [ 46 ].

also in 111,027 other papers