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

Confounding data from a Pfizer clinical trial has rattled the field of gene therapy for Duchenne muscular dystrophy, raising more questions about the regulatory standard used to approve a ...

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

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