α-<scp>d</scp>-Gal-cyclophellitol cyclosulfamidate is a Michaelis complex analog that stabilizes therapeutic lysosomal α-galactosidase A in Fabry disease

Abstract

α-<sc>d</sc>-Gal-cyclophellitol cyclosulfamidate is a new class of neutral, conformationally-constrained competitive glycosidase inhibitor that stabilizes α-gal A and prevents its degradation both <italic>in vitro</italic> and <italic>in cellulo</italic> by mimicry of the Michaelis complex conformation.