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.

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.

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.

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 ...

Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder ...