Quest Magazine: Dysfunction of Motor Circuits May Underlie SMA

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).

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.

Read the rest.

Click here for version in Spanish.

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