Non-iminosugar
glucocerebrosidase small molecule chaperones†
Juan Jose Marugan,Wenwei Huang,Omid Motabar,Wei Zheng,Jingbo Xiao,Samarjit Patnaik,Noel Southall,Wendy Westbroek,Wendy A. Lea,Anton Simeonov,Ehud Goldin,Maria A. DeBernardi,Ellen Sidransky
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.