The Collaborative Trajectory Analysis Project (cTAP) announces the publication of a novel, validated prognostic score for predicting the loss of ambulation (LoA) in patients with Duchenne muscular ...

In an eagerly anticipated decision, the Food and Drug Administration Thursday approved the first gene therapy for muscular dystrophy. "Today's approval addresses an urgent unmet medical need and is an ...

WASHINGTON (AP) — Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy. Sarepta reported the ...

Consensus directed medical therapy (CDMT) was used in 73.1% of DMD patients with moderate or severe left ventricular dysfunction, leaving nearly 30% without recommended treatment. Target doses were ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. The young ...

Learn more Servier has signed an agreement to acquire Edgewise Therapeutics’ muscular dystrophy business in a deal valued at up to $2.65bn. The value includes a $1.55bn upfront payment and as much as ...

FILE - The U.S. Food and Drug Administration campus in Silver Spring, Md., is photographed, Oct. 14, 2015. (AP Photo/Andrew Harnik, File) (Andrew Harnik, Copyright ...