Pharmacology & ToxicologybioRxiv
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…·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.
DOI 10.1242/dmm.040584