Spinal muscular atrophy (SMA) is a genetic condition that is passed down through families. It leads to muscle weakness, which may affect everyday functions such as head movement, sitting without help, ...

Scotland is now screening every newborn for a condition that can kill within two years. Here is what parents across the UK need to know.

Spinal muscular atrophy is a genetically inherited disorder that causes muscle weakness. Adults can get spinal muscular atrophy, but it’s rare in adults and progresses slowly. It doesn’t typically ...

The FIREFISH study showed that most children with type I spinal muscular atrophy (SMA) maintained or improved motor functions and feeding abilities over 5 years when treated with risdiplam. At the ...

Risdiplam, an oral pre–messenger RNA splicing modifier, is an efficacious treatment for persons with symptomatic spinal muscular atrophy (SMA). The safety and efficacy of risdiplam in presymptomatic ...

Spinal bulbar muscular atrophy (SBMA) causes a loss of motor neurons in the spinal cord and brainstem. It mainly affects facial and swallowing muscles and the muscles in the arms and legs. Previously, ...

Spinraza, also known as nusinersen, is a medicine used to treat spinal muscular atrophy (SMA) in children and adults. SMA is a genetic disease that is passed down through families. It leads to muscle ...

Distal spinal muscular atrophy (DSMA) is a rare genetic disease that causes a loss of muscle movement. It affects muscles further away from the center of the body, such as the hands, feet, and legs.

The risk of scoliosis was largely related to what type of SMA a patient had, with those with type III having the lowest risk and those with type II developing scoliosis at a later age than those with ...