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

• Главные авторы: Sangeetha Iyer (Автор), Feba S. Sam (Автор), Nina DiPrimio (Автор), Graeme Preston (Автор), Jan Verheijen (Автор), Kausalya Murthy (Автор), Zachary Parton (Автор), Hillary Tsang (Автор), Jessica Lao (Автор), Eva Morava (Автор), Ethan O. Perlstein (Автор)
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• Опубликовано: The Company of Biologists, 2019-11-01T00:00:00Z.
• Предметы: article
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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...