Pompe disease is a lysosomal storage disease (LSD) caused by a deficiency in the 
lysosomal enzyme acid alpha-glucosidase. In several LSDs, enzyme inhibitors have 
been used as small molecule chaperones to facilitate and increase the 
translocation of mutant protein from the endoplasmic reticulum to the lysosome. 
Enzyme activators with chaperone activity would be even more desirable as they 
would not inhibit the enzyme after translocation and might potentiate the 
activity of the enzyme that is successfully translocated. Herein we report our 
initial findings of a new series of acid alpha-glucosidase activators.