To help neurologists, clinicians and families understand the current evidence for a new gene therapy for Duchenne muscular ...

His previous roles at Zafgen, Inc., Biogen, Wyeth Biotech, Pfizer Inc., and Genetics Institute ... its Phase 2 CONNECT2-EDO51 study for Duchenne muscular dystrophy (DMD) to focus on data from ...

His previous roles at Zafgen, Inc., Biogen, Wyeth Biotech, Pfizer Inc., and Genetics Institute involved significant ... PepGen has temporarily paused its Phase 2 CONNECT2-EDO51 study for Duchenne ...

Gene therapy could be the newest breakthrough for Duchenne muscular dystrophy. While it may not be a cure, it can give independence to boys diagnosed with DMD.

Elevidys is the gene therapy product for Duchenne muscular dystrophy (DMD) and is designed to address the underlying cause of Duchenne through targeted skeletal, respiratory and cardiac muscle ...

Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular dystrophy (DMD), setting it on course to become the first non-steroidal ...

Pfizer has axed trials of its domagrozumab antibody for Duchenne muscular dystrophy (DMD), after it failed against efficacy targets. Pfizer has been evaluating domagrozumab under the codename PF ...

Delandistrogene moxeparvovec-rokl (ELEVIDYS) showed significant motor function improvements in 8- to 9-year-old Duchenne muscular dystrophy patients in part 2 of the phase 3 EMBARK study. In part 2 of ...

Multiple first-in-human trials showed functional and biomarker improvements in early data reported at ASGCT's annual meeting.