Mutations in the gene encoding dystrophin cause X-linked Duchenne muscular dystrophy (DMD), a devastating disease characterized by progressive muscle weakness, leads to cardiac or respiratory ...

This dystrophin transcript, theoretically, would exist in both muscle and the central nervous system, resulting in Duchenne muscular dystrophy in the former and neuronal dysfunction in the latter ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24 ... new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments ...

A Leicestershire man living with a rare muscle condition has revealed how gaming provides him with the "freedom" to live a ...

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

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

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

Duchenne muscular dystrophy (DMD) is an inherited muscle ... is a gene that produces a short protein that resembles dystrophin. It is injected in one IV infusion. There is no cure for DMD.

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

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

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