Children with severe SMA have substantial loss of motor and sensory neurons in the thoracic region of the spinal cord, a ...

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 ...

Taldefgrobep alfa did not significantly improve motor function across the total population of treated spinal muscular atrophy (SMA) patients in a pivotal Phase 3 clinical trial, but significant ...

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 ...