An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing severe muscle degeneration, also profoundly affects the brain, leading to ...

Dec. 16, 2024 — A novel drug holds promise for treating Duchenne muscular dystrophy (DMD), a rare genetic disorder that causes severe muscle degeneration. Researchers have discovered that an ...

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%.

Tom was recognised for his outstanding and creative fundraising achievements last year, raising money and awareness for Muscular Dystrophy UK, the leading charity for more than 110,000 people in ...

A PhD student with limb girdle muscular dystrophy will be the official race starter for this year's Oxford 10k.

Advanced imaging and theoretical physics have revealed how nanoclusters of the protein emerin form in cells, offering insights into potential treatments for Emery-Dreifuss muscular dystrophy (EDMD).

The study uncovers the molecular 'rules' driving the arrangement of emerin into nanoclusters and the mechanisms leading to their defective assembly in people with muscular dystrophy. A new ...

An inspirational Stirling schoolboy has been hailed for his bravery in battling a rare lifelong condition. Children and staff at St Margaret’s Primary, in Cowie, have been rallying round P7 ...

Pupils and staff visibly showed their support on the day by ‘going orange’, the brand colour of charity Muscular Dystrophy UK (MDUK) as part of a fundraising effort. Somhairle’s dad Gerry ...

Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...