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.

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

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.

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 ...

Since July, my family has been converting the garage space in our new house into my own bedroom, bathroom, kitchen, and medical supply room. We’ve faced one challenge after another as we prepare to ...

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

Halsey Blocher is a young woman who was diagnosed with SMA Type 1 in 1999 after a blood test revealed a deletion of the survival motor neuron gene. She now lives with her loving family in Fort Wayne, ...

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, ...