Riluzole attenuates spinal muscular atrophy disease progression in a mouse model.

摘要: Spinal muscular atrophy (SMA) is a motor neuron disease caused by mutations of the survival 1 gene (SMN1). No curative treatment available. Mutant mice carrying homozygous deletion Smn exon 7 directed to neurons display degenerative process similar that found in human SMA. To test whether riluzole, which exhibits neurotrophic properties, might have protective role SMA, mutant were treated with it after onset process. Riluzole improved median and exerted effect against aberrant cytoskeletal organization synaptic terminals but not loss proximal axons. These results demonstrate course SMA can be attenuated neuromuscular defects may warrant further investigation therapeutic trial