REGENXBIO Inc. (Nasdaq: RGNX) today announced it initiated enrollment in a new cohort of patients ages 1-3 in its Phase I/II AFFINITY DUCHENNE® trial to evaluate the safety and efficacy of RGX-202 in boys with Duchenne muscular dystrophy (Duchenne).
Sarepta stock rocketed Friday after hitting a "grand slam" approval that expands the market for its Duchenne muscular dystrophy gene therapy more than tenfold, according to one estimate.
Following the label expansion, Sarepta's (SRPT) Elevidys is approved to treat all DMD patients aged four years and older, regardless of ambulation status.