Atossa Therapeutics Receives FDA Orphan Drug Designation for (Z)-Endoxifen for the Treatment of Duchenne Muscular Dystrophy

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

Breakthrough in RNA Research Could Lead to Treatment for Neuromuscular Disorders

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 ...

Santhera Receives Swissmedic Approval of AGAMREE® (Vamorolone) for the Treatment of Duchenne Muscular Dystrophy

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

Regenxbio Shares Rise in Premarket As Duchenne Gene Therapy Shows Treatment Benefit

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 ...

Nxera Pharma Licenses Japan and Select Asia-Pacific Rights to Vamorolone for Duchenne Muscular Dystrophy from Santhera Pharmaceuticals

Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and ChinaNew ...
News9

New muscular dystrophy treatment gives family hope

Muscular dystrophy, known as DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe and live independently. Until now, there have ...
Benzinga.com

Solid Biosciences Positioned For Next-Gen Duchenne Muscular Dystrophy Treatment Success: Analyst

Wedbush initiated coverage on Solid Biosciences, Inc. (NASDAQ:SLDB), a gene therapy company focused on treating Duchenne muscular dystrophy (DMD). DMD is a genetic disease that causes progressive ...
Nasdaq

Edgewise Therapeutics to Present on Sevasemten for the Treatment of Becker Muscular Dystrophy at the 2025 MDA Clinical and Scientific Conference

– Company to host an Industry Forum to discuss the lived experience of Becker and clinical advancements featuring a leading neuromuscular disease expert and a patient advocate – Edgewise is sponsoring ...
Healio

Cell-based therapies, enzyme inhibitors at vanguard of muscular dystrophy treatment

Please provide your email address to receive an email when new articles are posted on . New therapies for muscular dystrophy must go beyond traditional corticosteroid administration. Treatments that ...
MedPage Today

Cardiac Complications of Duchenne Muscular Dystrophy

Cardiac complications in Duchenne muscular dystrophy (DMD) are the result of cardiac muscle involvement that accompanies the deterioration of skeletal muscle in the disease. The use of ...

Santhera Pharmaceuticals Holding AG: Santhera Receives Swissmedic Approval of AGAMREE (Vamorolone) for the Treatment of Duchenne Muscular Dystrophy

Commercial launch in Switzerland anticipated in H2 2026 Pratteln, Switzerland, January 15, 2026 - Santhera Pharmaceuticals (SIX: SANN) announces that the Swiss Agency for Therapeutic Products (Swissme ...