While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, ...
The biotech reported 5.5% mean absolute unadjusted dystrophin in its analysis of nine ambulatory boys with exon 53 DMD after 24 weeks of treatment. Wave was targeting expression levels greater ...
DMD is a genetic disease that causes progressive muscle weakness and degeneration caused by a mutation in the dystrophin gene. It’s the most common inherited neuromuscular disease and affects ...