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

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

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

Apitegromab is an investigational treatment aimed at improving motor function for people living with spinal muscular atrophy. Scholar Rock said it has also submitted and received validation for its ...

Spinal muscular atrophy (SMA) is a group of genetic disorders that causes a loss of motor nerve cells and muscle atrophy. There are five types of SMA relating to age of onset and symptom severity.

—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 ...

In a groundbreaking pilot study, researchers have found evidence that spinal cord stimulation could potentially benefit individuals with neurodegenerative diseases like spinal muscular atrophy (SMA).

Spinal muscular atrophy (SMA) is a rare genetic disorder in which the onset of progressive neurodegeneration begins before birth, owing to loss of the protein SMN. Drugs such as risdiplam ...

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