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 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 ...

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

Muscular dystrophy is a set of genetic diseases that cause muscles to become progressively weaker. Many forms of the disease ...

Nov. 13 -- THURSDAY, Nov. 12 (HealthDay News) -- Researchers report that a drug used to treat pneumonia might serve as an effective treatment against a type of muscular dystrophy. They tested the drug ...

Myotonic dystrophy type 1 (DM1) is the most common form of adult-onset muscular dystrophy, affecting about 1 in 8,000 people.

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

Grants include research funding in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth disease (CMT), Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy (FSHD), mitochondrial ...

Muscular dystrophy is a group of disorders that involve a progressive loss of muscle mass and consequent loss of strength. In general, the condition is caused by genetic variations that interfere with ...

In the spirit of the holidays, we're looking back on the time the Grateful Dead played a surprise set for kids with muscular ...