Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne, commemorated its 30th ...

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

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

Milestone represents a significant step forward in the development of deramiocel for the treatment of Duchenne muscular ...

Capricor Therapeutics (NASDAQ:CAPR) announced Wednesday that the European Medicines Agency has issued Orphan Drug and ...

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

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 ...

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

Throughout 2024, the FDA issued new drug approvals and expanded treatment indications across various rheumatologic diseases.

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

It’s easy for those who play or watch the best movies based on video games to want to be immersed in that fictional world.

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 ...