Bioevaluation of sixteen ADMDP stereoisomers toward alpha-galactosidase A: Development of a new pharmacological chaperone for the treatment of Fabry disease and potential enhancement of enzyme replacement therapy efficiency

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Bioevaluation of sixteen ADMDP stereoisomers toward alpha-galactosidase A: Development of a new pharmacological chaperone for the treatment of Fabry disease and potential enhancement of enzyme replacement therapy efficiency

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作者：Wei-Chieh Cheng^a ; ^b ; ^{wcheng@gate.sinica.edu.tw" class="auth_mail" title="E-mail the corresponding author} ; Jen-Hon Wang^a ; ^b ; Huang-Yi Li^a ; Sheng-Jhih Lu^a ; Jia-Ming Hu^a ; Wen-Yi Yun^a ; Cheng-Hsin Chiu^a ; Wen-Bin Yang^a ; Yin-Hsiu Chien^c ; Wuh-Liang Hwu^c
关键词：Fabry disease ; Lysosomal α-galactosidase A ; Lysosomal storage diseases ; Enzyme replacement therapy ; Pharmacological chaperone ; Pyrrolidine-based iminosugar
刊名：European Journal of Medicinal Chemistry
出版年：2016
出版时间：10 November 2016
年：2016
卷：123
期：Complete
页码：14-20
全文大小：1190 K

文摘

•: Inhibition study of ADMDP stereoisomers against α-Gal A is described.
•: 3-Epimer ADMDP is a new pharmacological chaperone for Fabry disease.
•: 3-Epimer ADMDP has a potential to enhance enzyme replacement therapy efficiency.