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

[Display omitted] •A short and high yielding route enables access to N-substituted amino(hydroxymethyl)pentanetriols.•Final products are potent inhibitors of a panel of β-galactosidases.•New compounds are efficient pharmacological chaperones for GM1-gangliosidosis-related human lysosomal β-galactosidase. N-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.