An experimental RNA therapy from Avidity Biosciences has early clinical trial results showing it reduced by half the expression of a gene at the root of a rare, inherited form of muscular dystrophy ...

Davia Jones said it took seven years for her son, Kyrie, to be diagnosed with Duchenne Muscular Dystrophy, a progressive ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which ...

Sarepta Therapeutics has come under pressure following the deaths of three patients using its treatments.

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 experimental compound ...

A retrospective cohort study found that, among 459 patients with DMD, constipation (32.5%) was the most common GI complication, followed by dysphagia (9.4%). Older age, lower body mass index, and use ...