The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but ...

REGENXBIO Inc.  today reported new, positive interim data from two additional patients in the Phase I/II portion of the AFFINITY DUCHENNE® trial of RGX-202, a differentiated investigational gene ...

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...