Gene activity in SMA may be dysregulated, but the way the disease affects genes seems to vary across types of tissues, per a ...

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 ...

Gene therapy that selectively delivers the SMN1 gene to the liver reduced alterations related to spinal muscular atrophy (SMA) in a mouse model, restoring muscle size and correcting pancreatic issues.