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...

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Detalles Bibliográficos
Autores principales: Iyer, Sangeetha, Sam, Feba S., DiPrimio, Nina, Preston, Graeme, Verheijen, Jan, Murthy, Kausalya, Parton, Zachary, Tsang, Hillary, Lao, Jessica, Morava, Eva, Perlstein, Ethan O.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6899038/
https://www.ncbi.nlm.nih.gov/pubmed/31636082
http://dx.doi.org/10.1242/dmm.040584

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6899038/
https://www.ncbi.nlm.nih.gov/pubmed/31636082
http://dx.doi.org/10.1242/dmm.040584

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