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 ...
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, ...
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, ...
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 ...
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 ...
BridgeBio Pharma will seek US approval of its limb-girdle muscular dystrophy (LGMD) small molecule after the drug met its ...
New research has identified the enzyme glutamate dehydrogenase 1 (GLUD1) as a new therapeutic target for Duchenne muscular dystrophy (DMD). In preclinical DMD mouse models, investigators demonstrated ...
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.
Some results have been hidden because they may be inaccessible to you
Show inaccessible results
Feedback