(5aR)-5a-C-Pentyl-4-epi-isofagomine: A powerful inhibitor of lysosomal β-galactosidase and a remarkable chaperone for mutations associated with GM1-gangliosidosis and Morquio disease type B
The synthesis of 3 new C-alkylated derivatives of 4-epi-isofagomine is described. Their inhibitory activities and selectivity towards human β-galactosidase were evaluated. Compound 1 behaved as an effective pharmacological chaperone towards several mutations.