Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for ...

MDA Legacy Award for Achievement in Clinical Research to honor Michio Hirano, MD, Columbia University Irving Medical Center ...

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE, /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) ("Atossa ...

Designation further supports (Z)-Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation SEATTLE ...

At the 44th Annual J.P. Morgan Healthcare Conference in early January 2026, Edgewise Therapeutics outlined its 2026 clinical ...

Commercial launch in Switzerland anticipated in H2 2026Pratteln, Switzerland, January 15, 2026 – Santhera Pharmaceuticals (SIX: SANN) announces ...

Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and ChinaNew ...

Researchers from Carnegie Mellon University have discovered a way to target RNA that could lead to new treatment options for myotonic dystrophy type 1 (DM1), the most common adult-onset form of ...

WILMINGTON, N.C. (WECT) -New Hanover County Fire Rescue firefighters are collecting donations at intersections around ...

Sarepta Therapeutics saw its stock skid 11% after the company announced preliminary unaudited 2025 sales results that missed analyst forecasts for its marketed Elevidys® (delandistrogene ...

Shares of  Regenxbio rose Monday in premarket trading after the company said its treatment for Duchenne muscular dystrophy delivered positive trial results, strengthening the case for a speedier ...

Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.