Highlights from the 2025 Muscular Dystrophy Association (MDA) Conference include clinical updates, expert insights, and ...

Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

This announcement follows a bipartisan letter that Senator Collins and a group of nine members of Congress sent last month to HHS Secretary Robert F. Kennedy Jr., requesting that Duchenne muscular ...

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 ...

HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...

Muscular dystrophy is a type of disease that causes the muscles in your body to lose strength and mass. As your muscles become weaker over time, it may be hard for you to do normal activities.

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

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 ...

A 5-year-old British boy suffering from the rare disease spinal muscular atrophy (SMA) has miraculously started walking four years after receiving the world’s most expensive gene therapy. On the 31st, ...

The muscular dystrophies represent disorders of progressive muscular degeneration and weakness. As a group, they exhibit clinical heterogeneity that reflects diverse molecular mechanisms responsible ...

Muscular dystrophies represent a group of inherited primary diseases of muscle, characterized by muscle fiber degeneration and muscle weakness. Classification of these conditions has traditionally ...