Rare neuromuscular diseases often lack treatments because developing targeted drugs is slow, costly and risky for companies. A new approach using AI and stem cell models could finally shift the ...

Primary endpoint is rise from floor (RFF) velocity with multiple secondary endpoints to assess muscle and pulmonary function ...

Because researchers have made such striking progress in developing drugs to treat neuromuscular diseases, Scott Delp, Ph.D., was surprised to learn that scientists conducting clinical trials were ...

The rare disorder Kennedy’s disease leads to progressively worsening muscle weakness in the limbs as well as difficulty speaking and swallowing. There are no FDA-approved treatments for the inherited ...

The recent approval of Regeneron’s Otarmeni underscores the maturation of gene therapies across a range of diseases. Here, ...

WALTHAM, Mass., May 20, 2026 (GLOBE NEWSWIRE) -- (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, ...

Grey’s Anatomy alum Steven W. Bailey has been diagnosed with a rare neuromuscular disorder known as Congenital Myasthenia Syndrome (CMS). Bailey, 54, explained via X on Friday, January 2, that he has ...

(Reuters) -FibroGen Inc's treatment for patients with a muscle-wasting disorder that typically binds them to a wheelchair failed to improve measures of upper limb strength, sending the drug ...

SAVANNAH, Georgia — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of patients is crucial, even at younger ages, a neurologist told an audience of ...

Sad woman sitting in front of cabinet in the dark. In a retrospective analysis, researchers characterized the prevalence and outcomes of neuropsychiatric conditions among patients hospitalized with ...