Rapid preparation of (3R,4S,5R) polyhydroxylated pyrrolidine-based libraries to discover a pharmacological chaperone for treatment of Fabry disease

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• 作者：Wei-Chieh Cheng^a ; ^b ; ^{wcheng@gate.sinica.edu.tw} ; Jen-Hon Wang^a ; ^b ; Wen-Yi Yun^a ; Huang-Yi Li^a ; Jia-Ming Hu^a
• 关键词：Azasugar ; Polyhydroxylated pyrrolidine ; Fabry disease ; Pharmacological chaperone ; Combinatorial chemistry ; Lysosomal α-galactosidase
• 刊名：European Journal of Medicinal Chemistry
• 出版年：2017
• 出版时间：27 January 2017
• 年：2017
• 卷：126
• 期：Complete
• 页码：1-6
• 全文大小：1070 K
• 卷排序：126

文摘

New pyrrolidine-based pharmacological chaperones for Fabry disease are developed. Compound 8 is a potent and selective pharmacological chaperone for Fabry disease. It is a durable and non-substrate competitive pharmacological chaperone.