Researchers have worked out the exact function of an enzyme that is critical for normal muscle structure and is involved in several muscular dystrophies. The findings could be used to develop rapid, ...

A novel potential therapy based on a natural human protein significantly slows muscle damage and improves function in mice who have the same genetic mutation as boys with the most common form of ...

Please provide your email address to receive an email when new articles are posted on . Viltepso, an injection treatment for Duchenne muscular dystrophy in patients with a confirmed mutation, ...

Evrysdi, a survival motor neuron 2-directed RNA splicing modifier, both sustained and improved motor function at 24 months in children and adults with type 2 or type 3 spinal muscular atrophy, ...

Muscle tissue provides the largest store of potentially available protein in the body; it is considerable reduced in children with severe protein energy malnutrition (PEM). After nutritional recovery ...

New research has demonstrated that a combined regimen of growth hormone and testosterone is safe, well-tolerated, and is ...

A team of Vanderbilt researchers has developed a wirelessly activated device that mimics the wavelike muscular function in the esophagus and small intestine responsible for transporting food and ...

Some patients with later-onset spinal muscular atrophy (SMA) type 2 and type 3 had improved motor function when the investigational monoclonal antibody apitegromab was added to their treatment, the ...

In Part 2 of the trial, patients 2-25 years of age received either risdiplam for 24 months or placebo for 12 months followed by risdiplam for 12 months. Treatment with risdiplam was associated with ...

The least-squares mean difference in the Hammersmith Functional Motor Scale-Expanded change from baseline at 12 months was 1.8 points for those 2 to 12 years receiving apitegromab vs placebo.