The US HHS has added Duchenne muscular dystrophy and metachromatic leukodystrophy to newborn screening, citing benefits of early detection and access to FDA-approved treatments.

This announcement follows a bipartisan letter that Senator Collins and a group of nine members of Congress sent last month to ...

The Muscular Dystrophy Association (MDA) today announced more than $2.7 million in new research grants to advance groundbreaking discoveries across multiple neuromuscular diseases, including ...

Cure Rare Disease (CRD) announced it has been awarded a $7.4 million grant from the California Institute for Regenerative Medicine (CIRM) to advance the development of a novel gene therapy for ...

In November 2025, Epicrispr Biotechnologies Inc. announced a clinical trial is to learn how safe and tolerable EPI-321 is and ...

Webinar to be held Wednesday, December 17, 2025, at 1:00 p.m. ET –SAN DIEGO, Dec. 16, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics, Inc.

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 ...

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 ...

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 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 ...