Sustained exon skipping, dystrophin restoration, and improved muscle indicators were observed, including reduced inflammation and fibrosis. A 3.8-second improvement in time to rise (TTR ...

The drug – formerly known as SRP-5051 – is a more potent version of Exondys 51 (eteplirsen), an exon-skipping therapy that can be used to treat around 13% of the population with the muscle ...

The randomised, placebo-controlled, two-part, double-blind trial will assess the therapy in individuals with a DMD gene mutation amenable to exon 45 skipping ... oligomer (PMO) and claims to ...