For a boy with SMA in China, starting Evrysdi (risdiplam) just 12 days after birth was safe and helped him reach key motor ...

The evolution of functional assessments for spinal muscular atrophy (SMA) highlights the challenges of adapting pediatric scales for adults and the need for modified evaluations to accurately ...

Researchers studied 118 SMA type 2 patients and saw their RULM scores stayed higher than expected had their disease kept its ...

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 ...

In which young patients with spinal muscular atrophy will spinal braces and growing rods be most helpful? Ashley Webb, MD, assistant professor of pediatric neurology and neuromuscular program ...

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 University of Texas Health ...

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 ...