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 ...

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

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

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

Regenxbio shares surged 20% after disclosing new, positive efficacy and safety data of its Duchenne muscular dystrophy treatment. Shares were recently trading around $11.55. The stock is down 46% on ...

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

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 ...

DICKSON CITY, Pa. — The Eagle Hose Company in Dickson City on Saturday, November 16 hosted a benefit for the Hetzel family as ...

Disabled people give testimony in phase three of the Covid inquiry. The latest part of the Covid inquiry, which looks at the impact of the pandemic on the NHS, has heard powerful evidence from ...

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 ...

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