Duchenne muscular dystrophy (DMD) is a disorder that causes progressive muscle weakness and a loss of function. The symptoms appear early in life, and most affected individuals require the use of ...

After months of uncertainty and controversy, the FDA has given approval to Sarepta's Duchenne muscular dystrophy drug – even though it hasn't yet shown any proof it can slow progress of the ...

Recommendation is based on Phase 3 EPIDYS trial data that demonstrated Duvyzat (givinostat) provides statistically and clinically meaningful treatment benefits in individuals with Duchenne muscular ...

John Brandsema, MD, a pediatric neurologist at Children’s Hospital of Philadelphia, speaks with Healio about the latest ...

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

You can reach Jason on Signal at JasonMast.05. Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the stage for a new ...

Suneel Ram’s needs gradually became greater after he was diagnosed at age 3 with Duchenne muscular dystrophy, a muscle-wasting genetic disorder that afflicts one in 5,000 boys. Today ...

Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026.

(RTTNews) - Sarepta Therapeutics (SRPT) issued an update related to ELEVIDYS, the only approved gene therapy in patients with Duchenne muscular dystrophy. Following the safety update on acute ...

This story is republished from STAT, the health and medicine news site that’s a partner to the Globe. Sign up for STAT’s free Morning Rounds newsletter here. Wave Life Sciences’ experimental ...