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 ...

Regenxbio’s investigational gene therapy improved muscle function for patients with Duchenne muscular dystrophy (DMD) in a phase 1/2 study. | Regenxbio’s investigational gene therapy improved muscle ...

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

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

A small trial of an experimental vaccine has shown promising results for patients with the most aggressive type of breast ...

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

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

StitchR, a new gene therapy technique, delivers large genes in two parts to treat muscular dystrophies by restoring critical proteins in animal models.

The agency's openness to a targeted pivotal study shows it’s still willing to consider accelerated clearance for Duchenne ...

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...

StitchR makes it possible to deliver larger payloads with current vectors, expanding the list of diseases targetable by gene therapies.

Regenxbio reported Monday the first clinical evidence showing improved muscle function in boys with Duchenne muscular dystrophy following treatment with its experimental gene therapy. The positive ...