Coral Crespo Gómez

Pharmacological Activation of Autophagy Ameliorates Neuromuscular-Associated Defects and Extends Lifespan in a Caenorhabditis elegans Model of Spinal Muscular Atrophy

Findings support enhancing autophagic activity as a combinatorial treatment strategy for SMA patients by identifying that pharmacological activation of autophagy, but not inhibition, significantly improved neuromuscular function, as indicated by two independent functional readouts.