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.
WUSTL researchers have converted skin cells into motor neurons without going through the stem cell state. The new technique could help in the development of devastating neurodegenerative diseases, like ALS, that affect motor neurons.
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 report insufficient blood supply could contribute to motor neuron loss in SMA.
A new study uncovers molecular changes which may explain why motor neurons, rather than other neurons, are affected by the childhood illness, SMA.
Researchers identify Neurexin2 as a new target for potential treatment of spinal muscular atrophy.
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.