Zolgensma, an FDA approved gene therapy, supplements the production of the SMN protein to improve motor neuron function in children with spinal muscular atrophy after just one dose.
A new drug shows promise for improving outcomes for people with SMA.
A study suggests that spinal muscular atrophy (SMA), a genetic neuromuscular disease in infants and children, results primarily from motor circuit dysfunction, not motor neuron or muscle cell dysfunction, as is commonly thought. In a second study, the researchers identified the molecular pathway in SMA that leads to problems with motor function.
A new study implicates a key cellular mechanism as defective in SMA for the first time, providing a new lead for developing future interventions.
Researchers believe they have discovered a promising new treatment for spinal muscular atrophy, a leading genetic cause of death in children.
