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

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

A Prescription Drug User Fee Act (PDUFA) target action date for apitegromab has been set for Sept. 22, 2025.

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 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.

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 ...

22. 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 ...

Opens in a new tab or window How does one time surgery for scoliosis in children with pediatric spinal muscular atrophy get a maximum benefit without impeding growth? Dr. Ashley E. Webb of the ...