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

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

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

The Muscular Dystrophy Association (MDA) has selected Lily Sander from Charlotte, North Carolina, as its 2025 MDA National ...

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

Celebrating 75 Years of Progress, Legacy, Impact, and Momentum Leading up to Rare Disease Day on February 28 Muscular ...

Edgewise Therapeutics , Inc. (NASDAQ:EWTX), with a market capitalization of $2.67 billion, is a biopharmaceutical company focused on developing innovative therapies for severe, rare muscle disorders.

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

The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...

Discover the importance of vision, genetic eye disorders, and RNA-based therapies for precision treatment in India and ...

In year two of the trial, Elevidys demonstrated statistically significant and clinically improvements across three key ...