Risdiplam improves motor function in infants with spinal muscular atrophy

Until recently, babies and children with spinal muscular atrophy (SMA) lacked any type of treatment capable of stopping the neuromuscular disease from progressing. In SMA, damaged motor neurons in the spinal cord fail to correctly send messages to the muscle cells. As a result, infants and children with SMA may not be able to hold their head up, sit, or walk, depending on the severity of their mutation.

source https://medicalxpress.com/news/2021-08-risdiplam-motor-function-infants-spinal.html