Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG

Repurposing the aldose reductase inhibitor and diabetic neuropathy drug epalrestat for the congenital disorder of glycosylation PMM2-CDG

Phosphomannomutase 2 deficiency, or PMM2-CDG, is the most common congenital disorder of glycosylation and affects over 1000 patients globally. There are no approved drugs that treat the symptoms or root cause of PMM2-CDG. To identify clinically actionable compounds that boost human PMM2 enzyme funct...

Full description

Bibliographic Details
Main Authors: Sangeetha Iyer, Feba S. Sam, Nina DiPrimio, Graeme Preston, Jan Verheijen, Kausalya Murthy, Zachary Parton, Hillary Tsang, Jessica Lao, Eva Morava, Ethan O. Perlstein
Format: Article
Language:English
Published: The Company of Biologists 2019-11-01
Series:Disease Models & Mechanisms
Subjects:
phosphomannomutase 2 deficiency
drug repurposing
pmm2-cdg
congenital disorder of glycosylation
aldose reductase inhibitor
epalrestat
Online Access:http://dmm.biologists.org/content/12/11/dmm040584

Internet

http://dmm.biologists.org/content/12/11/dmm040584

Similar Items