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.

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

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

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 ...

Helen Baldwin lives in Jefferson, North Carolina, with her husband, Randy plus their two dogs, a cat, and untold wild critters outside. Their firstborn’s family, including two delightful children, ...

Some 70% of children with spinal muscle atrophy (SMA) type 1 who underwent treatment with Spinraza alone or with Spinraza and then Zolgensma reached some motor milestones over 18 months, with greater ...