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

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

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

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 Inc. (Nasdaq: RGNX) today announced that AFFINITY DUCHENNE®, the multi-center, open-label trial of RGX-202, a ...

In mice with muscular dystrophy, the treatment restored large therapeutic muscle proteins to normal levels, the researchers ...

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 ...

After winning an expanded approval for its gene therapy to treat Duchenne muscular dystrophy earlier this year, Sarepta ...

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

Sales of Elevidys are quickly gaining ground on Sarepta's exon-skipping trio, which need to be dosed chronically. In the ...

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

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