RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, ...

ROCKVILLE, MD, USA I March 19, 2025 I REGENXBIO Inc. (Nasdaq: RGNX) today reported new, positive interim data from two additional patients in the Phase ...

A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...

Myotonic dystrophy type 1 (DM1) is the most common adult-onset form of muscular dystrophy and a condition that severely ...

Sarepta Therapeutics (SRPT) stock drops 25% as the company reports a patient death linked to Elevidys, its gene therapy developed with Roche (RHHBY). Read more here.