Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data ...

Dyne Therapeutics plans to submit its next-gen Duchenne muscular dystrophy drug to the FDA after a successful trial.

On some level, biology is all about regulation, regulation, regulation. One way that our cells regulate their genes is through a process called alternative splicing, which entails the selective ...

Duchenne muscular dystrophy (DMD) is a neuromuscular disorder that results from mutations in the DMD gene. Gene therapies for DMD change genetic material in a person’s body to treat this condition.

Dyne Therapeutics, citing trial data, said it will ask the FDA to approve DYNE-251, its therapy for DMD amenable to exon 51 skipping.

KIT-exon-11 mutations in GISTs correlate with higher partial response rates and improved surgical outcomes with neoadjuvant Gleevec. Non-KIT exon 11 mutations and wild-type KIT/PDGFRA GISTs show less ...