Non-iminosugar glucocerebrosidase small molecule chaperones.

Therapeutic Approaches
Medicinal Chemistry


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.


Marugan, Juan Jose; Huang, Wenwei; Motabar, Omid; Zheng, Wei; Xiao, Jingbo; Patnaik, Samarjit; Southall, Noel; Westbroek, Wendy; Lea, Wendy A; Simeonov, Anton; Goldin, Ehud; Debernardi, Maria A; Sidransky, Ellen;

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