Dyne Therapeutics Announces Submission of Biologics License Application (BLA) to U.S. FDA for Z-Rostudirsen in Exon 51 Duchenne Muscular Dystrophy (DMD)
Dyne Therapeutics has submitted a Biologics License Application to the U.S. FDA for accelerated approval of z-rostudirsen (DYNE-251), a treatment targeting Duchenne Muscular Dystrophy patients amenable to exon 51 skipping. The submission is based on significant results from the DELIVER trial showing increased dystrophin production and functional im…