Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...
Jeffrey Del Carmen, Chief Commercial Officer of Catalyst Pharmaceuticals ( CPRX +0.06%), exercised and immediately sold ...
(Reuters) - Stealth BioTherapeutics Corp's shares crashed on Friday after its lead neuromuscular disorder drug candidate failed to help patients walk better and reduce fatigue, a major setback to the ...
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 ...
—Children with neuromuscular diseases and cerebral palsy often report disrupted sleep, which is usually attributed to breathing disorders. But new research points to a new potential contributor: ...
Grants include research funding in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), mitochondrial ...
Using two smartphone cameras and a free video app, a detailed intervention can be produced with accuracy for diagnosing neuromuscular diseases. The innovative discovery was made by researchers from ...
BOSTON, November 24, 2025--(BUSINESS WIRE)--EMD Serono, the Healthcare business of Merck KGaA, Darmstadt, Germany, in the U.S. and Canada, today announced the U.S. Food and Drug Administration (FDA) ...
Scientists have created a bioengineered 3-D model that mimics the biology of two rare diseases. The researchers used the 'tissue chip' model to show how a drug could potentially treat the diseases, ...
This year marks the tenth anniversary of the TREAT-NMD Advisory Committee for Therapeutics (TACT), a group of multidisciplinary experts that evaluates drug development programmes for rare ...
MANILA (Reuters) - Philippine President Rodrigo Duterte said he has a chronic neuromuscular disorder called myasthenia gravis that is causing one of his eyelids to droop. Duterte made the comment ...