Animal studies suggest that spinal muscular atrophy may result primarily from motor circuit dysfunction, not motor neuron or muscle cell dysfunction, as is commonly thought
Results from a study in fruit flies conducted by scientists in the Motor Neuron Center at Columbia University Medical Center in New York suggest that spinal muscular atrophy (SMA) — commonly thought to be a disease of muscle-controlling nerve cells calledmotor neurons — instead results from the dysfunction of motor circuits (networks made up of different types of specialized neurons that coordinate muscle movement).
A phase 2-3 clinical trial based on the findings is testing whether an existing drug called dalfampridine can improve walking ability and endurance in adults with type 3 SMA. (Dalfampridine is marketed under the name Ampyra for treatment of multiple sclerosis.)
In a second study, researchers identified the molecular pathway in SMA that leads to problems with motor function.
The findings could point the way to new therapeutic strategies for SMA.
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