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

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

Enlarged calves and progression of the pelvifemoral ... phosphokinasemia have been shown to occur in the Duchenne type of muscular dystrophy before any detectable signs of muscle weakness, 3 ...

Specific mutations in the DMD gene influence how long patients retain the ability to walk, even when treated with ...

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 ...

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

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

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 ...

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