Second-generation iminoxylitol-based pharmacological chaperones for the treatment of Gaucher disease.


A series of O-alkyl iminoxylitol derivatives was synthesized and evaluated as β-glucocerebrosidase (GCase) inhibitors. This structure-activity study shows a dramatic influence of the position of the alkyl chain (α-C1, O2, O3, or O4) on human GCase inhibition. Remarkably, 1,2-shift of the alkyl chain from C1 to O2 was found to maintain high inhibitory… (More)
DOI: 10.1002/cmdc.201000469


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