Intrathecal treatment with a vector-based gene therapy was associated with a greater improvement in motor function at 52 ...

A film about a Kent boy who lives with a rare condition has been nominated for a Bafta TV award. Sky's Disability & Me ...

The FDA has accepted for Priority Review the BLA for apitegromab for the improvement of motor function in patients with spinal muscular atrophy.

In the time since the Novartis gene therapy Zolgensma was approved for babies with spinal muscular atrophy, other treatments ...

Use precise geolocation data and actively scan device characteristics for identification. This is done to store and access ...

Since 2016, the FDA has approved three disease-modifying treatments for spinal muscular atrophy, with several ...

—Sleep-disordered breathing occurs frequently in patients with spinal muscular atrophy and can be obstructive or non-obstructive, with those diagnosed with non-obstructive SDB having worse ...

Video: Spinal Muscular Atrophy: When is Surgical Intervention Appropriate? —How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit ...

A rare genetic condition, Spinal Muscular Atrophy (SMA) affects one in 7,000 people in India. It mostly affects children and ...

The high court noted that SMA is a debilitating disease which has no cure, and the only approved drug, Risdiplam, marketed ...

A pair of clinical data sets gives Novartis confidence in a new formulation of the company’s gene therapy Zolgensma for the ...

for apitegromab for the improvement of motor functions in patients with spinal muscular atrophy (SMA). Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and ...