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

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 ...

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

Endoxifen program into rare pediatric neuromuscular disease along with previously received Rare Pediatric Disease Designation ...

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 ...

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 ...

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 ...

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 ...

– 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 ...

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 ...

Company CEO Doug Ingram said that the company “faced and overcame challenges in 2025.” ・Elevidys, a medicine intended for the ...