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

Columnist Alyssa Silva had an epiphany on a recent trip to Boston: She can battle her discouraging days by finding gratitude.

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

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

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

Individually and collectively, it’s vital to be sensitive to what’s happening in the world around us. This awareness allows ...

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

Since her SMA assignment, columnist Helen Baldwin has learned to look for the bright spots — even in Hurricane Helene's ...

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

Advanced genetic testing helped clinicians identify novel SMN1 mutations in two children in China, and correctly diagnose ...