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.

Individually and collectively, it’s vital to be sensitive to what’s happening in the world around us. This awareness allows us to be empathetic and strive for change that improves society for ...

Living with spinal muscular atrophy (SMA) often means facing many hardships and uncertainties. With physical limitations, emotional hurdles, and complex medical issues, SMA is constantly shaping and ...

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, ...

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 ...

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

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

Spinal muscular atrophy (SMA) is a neuromuscular disorder that causes progressive muscle weakness, which can lead to fatigue. Fatigue is an extreme tiredness or exhaustion that doesn’t go away with ...