Glucosylceramide synthase inhibitors and therapeutic methods using the same

摘要

Glucosylceramide synthase inhibitors and compositions containing the same are disclosed. Methods of using the glucosylceramide synthase inhibitors in the treatment of diseases and conditions wherein inhibition of glucosylceramide synthase provides a benefit, like Gaucher disease and Fabry disease, also are disclosed.

主权项

1. A compound having a structure wherein R1 is H or C1-3alkyl;
R2is  and R3 is wherein the fused ring B is a five- or six-membered ring, saturated or partially or fully unsaturated, comprising carbon atoms and one or two heteroatoms selected from O, S, and NRa, and wherein the phenyl ring is optionally substituted with one or two substituents selected from the group consisting of halo, C1-3alkyl, ORa, CO2Ra, halomethyl, halomethoxy, cyano, nitro, and N(Ra)2; or a pharmaceutically acceptable salt, solvate, hydrate, or prodrug thereof, and Ra independently is H or C1-3alkyl.