Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG
S. Iyer, F. S. Sam, N. DiPrimio, G. Preston, J. Verhejein, K. Murthy, Z. Parton, H. Tsang, J. Lao, E. Morava, E. O. Perlstein
This paper identifies epalrestat, an existing drug, as a PMM2 enzyme activator for PMM2-CDG, showing it improves enzyme activity in patient fibroblasts.
bioRxiv · Sep 26, 2019Pharmacology & Toxicology