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Issue 1, 2012
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Non-iminosugar glucocerebrosidase small molecule chaperones

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Abstract

Small molecule chaperones are a promising therapeutic approach for the Lysosomal Storage Disorders (LSDs). Here, we report the discovery of a new series of non-iminosugar glucocerebrosidase inhibitors with chaperone capacity, and describe their structure–activity relationship (SAR), selectivity, cell activity and phamacokinetics.

Graphical abstract: Non-iminosugar glucocerebrosidase small molecule chaperones

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Publication details

The article was received on 01 Aug 2011, accepted on 21 Sep 2011 and first published on 24 Oct 2011


Article type: Concise Article
DOI: 10.1039/C1MD00200G
Med. Chem. Commun., 2012,3, 56-60

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    Non-iminosugar glucocerebrosidase small molecule chaperones

    J. J. Marugan, W. Huang, O. Motabar, W. Zheng, J. Xiao, S. Patnaik, N. Southall, W. Westbroek, W. A. Lea, A. Simeonov, E. Goldin, M. A. DeBernardi and E. Sidransky, Med. Chem. Commun., 2012, 3, 56
    DOI: 10.1039/C1MD00200G

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