Guaiacol as a drug candidate for treating adult polyglucosan body disease

Adult polyglucosan body disease (APBD) is a late-onset disease caused by intracellular accumulation of polyglucosan bodies, formed due to glycogen-branching enzyme (GBE) deficiency. To find a treatment for APBD, we screened 1,700 FDA-approved compounds in fibroblasts derived from APBD-modeling GBE1-...

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Bibliographic Details
Main Authors: Kakhlon, O, Ferreira, I, Solmesky, L, Khazanov, N, Lossos, A, Alvarez, R, Yetil, D, Pampou, S, Weil, M, Senderowitz, H, Escriba, P, Yue, W, Akman, H
Format: Journal article
Language:English
Published: American Society for Clinical Investigation 2018