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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Dettagli Bibliografici
Pubblicato in:Dis Model Mech
Autori principali: Iyer, Sangeetha, Sam, Feba S., DiPrimio, Nina, Preston, Graeme, Verheijen, Jan, Murthy, Kausalya, Parton, Zachary, Tsang, Hillary, Lao, Jessica, Morava, Eva, Perlstein, Ethan O.
Natura: Artigo
Lingua:Inglês
Pubblicazione: The Company of Biologists Ltd 2019
Soggetti:
Research Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6899038/
https://ncbi.nlm.nih.gov/pubmed/31636082
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1242/dmm.040584
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