A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols

N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of beta-D-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for G(M1)-gangliosidosis-associated lysosomal acid beta-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease. (C) 2017 Elsevier Ltd. All rights reserved.