Patient advocate Austin Leclaire, who died Feb. 1, played a pivotal role in spurring the development of the first medicines ...

Cumberland Pharmaceuticals’ Duchenne muscular dystrophy (DMD) drug has improved the amount of blood pumped by the heart, ...

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Robert Davidson is the first UK patient recruited to a new medical trial looking to support children with heart failure ...

Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...

BOSTON - Entrada Therapeutics , Inc. (NASDAQ:TRDA) received authorization from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to initiate a Phase 1/2 clinical trial for its ...

Percheron Therapeutics says it will abandon its lead drug candidate altogether, following December's Duchenne muscular dystrophy trial flop.

While the last decade has brought considerable progress for patients with DMD, substantial unmet need remains. Several ...

Riding recent momentum in the Duchenne muscular dystrophy space, Capricor Therapeutics, Wave Life Sciences, Regenxbio and ...

At just 15 years old, Ragheeb has already made a tremendous impact on his community as an ambassador for Capes for Kids, an ...

Contrary to prior data, this study shows that neither eosinophils nor STAT6-mediated IL-4/IL-13 signaling contribute ...