This announcement follows a bipartisan letter that Senator Collins and a group of nine members of Congress sent last month to ...
HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...
Muscular Dystrophies are a group of genetic conditions characterized by muscle weaknesses. They are genetic conditions and have different sub-categories depending on which area of muscle mass is ...
Muscular Dystrophy (MD) is more than a medical condition—it's a journey that reshapes lives. Imagine trying to climb stairs, run, or even stand, only to find your muscles not cooperating. That’s the ...
Italfarmaco & JCR Pharma enter commercialisation agreement for givinostat to treat Duchenne muscular dystrophy in Japan: Milan, Italy Monday, December 29, 2025, 14:00 Hrs [IST] It ...
Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...
Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people. While it is well known for causing muscle weakness and stiffness, DM1 also ...
Grants include research funding in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), mitochondrial ...
ST. LOUIS — When families streamed into Forest Park Sunday morning, many of them were there for a walk to cover new ground in the fight against muscular dystrophy. "You know for me the diagnosis was ...
The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...
The powerful gene editing technology CRISPR is one small step closer to treating a human disease. In a new paper published in Science, researchers led by Eric Olson, professor and chair of molecular ...
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