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

• DOI: 10.1039/c9sc03342d
• Publication Date: 2019-08-20T07:46:58Z
• Authors: Marta Artola; Christinne Hedberg; Rhianna J. Rowland; Lluís Raich; Kassiani Kytidou; Liang Wu; Amanda Schaaf; Maria Joao Ferraz; Gijsbert A. van d...; Jeroen D. C. Codée; Carme Rovira; Johannes M. F. G....; Gideon J. Davies; Herman S. Overkleeft
• Keywords: 0301 basic medicine; 03 medical and health sciences; 3. Good health

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> cellulo</italic> by mimicry the Michaelis complex conformation.