Non-iminosugar glucocerebrosidase small molecule chaperones

Medchemcomm. 2012 Jan;3(1):56-60. doi: 10.1039/C1MD00200G. Epub 2011 Oct 24.


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.