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

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

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

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