Duchenne, one of the most severe forms of muscular dystrophy, affects about one out of every 3,600 boys. The inherited rare ...

The prevalence of Duchenne muscular dystrophy is 1 in 35,000 males. [12] The prevalence of pervasive developmental disorder in the male population is 1.6 in 1000, approximately four times more ...

The diagnosis of Duchenne muscular dystrophy was made in all patients on the basis of the clinical history and the absence or near-absence of the dystrophin protein on muscle biopsy. In addition ...

Based on the positive findings from the Phase 1/2 study, Genethon is planning to launch Phase 3 testing of GNT0004 next year.

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy ...

Gene therapy for some diseases, including Duchenne muscular dystrophy (DMD), can be tricky because the needed gene is often ...

Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy ... a protein called dystrophin that ...

Regenxbio has revealed plans to submit a biologics licence application (BLA) for RGX-202, its investigational gene therapy for Duchenne muscular dystrophy ... in the dystrophin protein that ...

Clinical development and trial testing of vesleteplirsen (SRP-5051) for Duchenne muscular dystrophy (DMD) was stopped, with ...

StitchR enabled expression of the protein Dysferlin, which is lacking in individuals with limb girdle muscular dystrophy type ...

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...