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

Dr. Michio Hirano to receive the MDA Legacy Award for Achievement in Clinical Research; Allison Moore to receive the MDA ...

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 ...

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 ...

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 ...

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

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 ...

A stretch of viral DNA in the mouse genome gives cells in early-stage embryos the potential to become almost any cell type in ...

In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid ...