The data come from the FORWARD-53 trial of WVE-N531, an exon-skipping oligonucleotide designed to treat boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. “Despite progress in ...

Wave’s DMD program, centered around WVE-N531, has shown promise as an alternative exon-skipping therapy. The company sees an opportunity in this space following Sarepta Therapeutics (NASDAQ:SRPT)’ ...

WVE-N531 demonstrated statistically significant improvements in muscle biopsy measures and functional measures in patients with Duchenne muscular dystrophy (DMD) in the phase 2 FORWARD-53 trial.