Children with severe SMA have substantial loss of motor and sensory neurons in the thoracic region of the spinal cord, a ...

Taldefgrobep alfa as an add-on therapy significantly improved motor function in pivotal Phase 3 trial, but racial differences ...

Years after losing a child to SMA, columnist Helen Baldwin shares that she has many blessings to be thankful for — courtesy ...

Liver-specific SMN1 gene therapy both improved liver functioning, and also reduced other SMA-like symptoms in a mouse model ...

Recent progress in SMA research and advocacy was celebrated at Cure SMA's annual Hope on the Hill event in Washington, D.C.

Muscle fibers in children with SMA type 1 show substantial differences in components of the myosin protein called heavy ...

Columnist Kevin Schaefer feels adulthood with SMA is like a "Star Trek" mission: to boldly go into uncharted territory.

Treatment with Spinraza (nusinersen) can help make walking less tiring over time for ambulatory patients with spinal muscular atrophy (SMA), a study reported. The study, “Performance fatigability in ...

More than half of SMA patients in an Australian survey said they did not notice any improvement in their symptoms following ...

SMA treatment Spinraza followed by Zolgensma improved motor function and prevented worsening of breathing difficulties in a ...