Sarepta says its Duchenne drug more effective than older med

Sarepta Therapeutics said on Monday a mid-stage trial showed its experimental drug produced higher levels of a specific protein deficient in some patients with Duchenne muscular dystrophy (DMD) than its older drug Exondys 51.

DMD, a muscle wasting disorder, is a rare genetic disorder that affects an estimated one-in-3,500 male births worldwide, and majority of the patients lack the dystrophin protein which keeps muscles intact.

The drug SRP-5051 and Exondys 51 both belong to a class of "exon-skipping" therapies, which work by skipping specific parts of genes, called exons, to allow the body to make shorter forms of the dystrophin protein.

Sarepta said the new drug, when given every four weeks, showed "substantially higher increases" in dystrophin and exon skipping compared to Exondys 51 given every week.

https://finance.yahoo.com/news/1-sarepta-says-experimental-duchenne-123733519.html