Keros Therapeutics, Inc. KROS is pushing its lead asset, rinvatercept, into neuromuscular disease with Duchenne muscular ...

Kids with Duchenne muscular dystrophy and their families deserve the chance to decide their own future.

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for high unmet need diseases ...

CEO Jasbir Seehra outlined the company’s development priorities and partnership plans during a presentation at Oppenheimer’s 36th Annual Healthcare Life Sciences conference, highlighting plans to init ...

Atossa's Rare Pediatric Disease Designation for (Z)-endoxifen in neuromuscular diseases qualifies for a future PRV upon FDA approval SEATTLE, Feb. 5, 2026 /PRNewswire/ -- Atossa Therapeutics, Inc.

NASHVILLE, Tenn., Feb. 4, 2026 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on developing new products for rare diseases, announced today ...

Daughter Verena shares her personal story of living with Limb-girdle type 2A muscular dystrophy. The progressive disease affects muscles around the shoulders, limbs, hips, and thighs, making simple ...

TEMPE, AZ (AZFamily) — An Arizona State University student is forging a new path of independence, all while raising awareness for those battling neuromuscular disabilities like himself. On Friday ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

ELLSWORTH, Maine (WABI) - On Saturday, November 22, those who head to The Grand Theater in Ellsworth will get a chance to see the state of Maine in a way they’ve likely never seen before. “Seasons of ...