Regenxbio is pushing its Duchenne muscular dystrophy gene therapy into pivotal development, with a BLA planned for ...

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

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

Specifically, StitchR restored the protein Dystrophin, which is absent in patients with Duchenne muscular dystrophy, and the ...

Regenxbio reported improved muscle function in boys with Duchenne muscular dystrophy using their gene therapy RGX-202. The ...

Based on a small trial with positive muscle performance data, Regenxbio is expected to pursue approval of its gene therapy ...

a potential best-in-class gene therapy for Duchenne muscular dystrophy, has advanced to pivotal stage and dosed its first patient. The company also announced new, positive efficacy and safety data ...

Put up against placebo in the phase 3 EMBARK trial, delandistrogene moxeparvovec (Elevidys) did not significantly improve ...

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

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size.

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

In-shoe sensors, AI and biomarkers will monitor muscular dystrophy and atrophy in children, adolescents and adults — with ...