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Synthesis of a New β-Galactosidase Inhibitor Displaying Pharmacological Chaperone Properties for GM1 Gangliosidosis
Clemente, Francesca; Martínez Bailén, Macarena; Matassini, Camilla; Morrone, Amelia; Falliano, Silvia; Caciotti, Anna; Paoli, Paolo; Goti, Andrea; Cardona, Francesca (MDPI, 2022)GM1 gangliosidosis is a rare lysosomal disease caused by the deficiency of the enzyme β-galactosidase (β-Gal; GLB1; E.C. ...