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Medchemcomm. 2012 Jan;3(1):56-60. Epub 2011 Oct 24.

Non-iminosugar glucocerebrosidase small molecule chaperones.

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NIH Chemical Genomics Center, National Human Genome Research Institute, National Institutes of Health, 9800 Medical Center Drive, Rockville, 20850, MD, USA.


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 phamacokinetics.

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