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

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.

And that was the last time I tried. When we were eight years old, we had severe scoliosis which caused us to have to get a spinal fusion. Unfortunately, one of the bad things about it is it ...

Apitegromab, a fully human monoclonal antibody, works by selectively binding to the pro- and latent forms of myostatin in order to inhibit myostatin activation.

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 ...

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).

Novartis AG (NYSE:NVS) announced safety and efficacy results from the Phase 3 program for investigational intrathecal ...

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 ...