Young children with severe spinal muscular atrophy (SMA) have substantial loss of motor neurons in the thoracic region of the ...

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

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

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

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 ...

At Cure SMA’s 13th annual Hope on the Hill event in Washington, D.C., spinal muscular atrophy (SMA) patients, their families, lawmakers, and other advocates gathered to celebrate recent progress in ...

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, ...