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 ...
Recent studies have defined a group of muscular dystrophies, now termed the dystroglycanopathies, as novel disorders of glycosylation. These conditions include Walker–Warburg syndrome, ...
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 ...
Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...
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 ...