Lucerastat

Description: Lucerastat is a very effective and selective alpha-gal A (alpha-D-galactosidase) inhibitor for the treatment of lipid storage diseases and Fabry disease. Target: Others. Smiles: CCCCN1C[C@H](O)[C@@H](O)[C@@H](O)[C@H]1CO. References: Takai T, Higaki K, Aguilar-Moncayo M, Mena-Barragán T, Hirano Y, Yura K, Yu L, Ninomiya H, García-Moreno MI, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y. A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis. Mol Ther. 2013 Mar,21(3):526-32. doi: 10.1038/mt.2012.263. Epub 2013 Jan 22. PubMed PMID: 23337983, PubMed Central PMCID: PMC3589148.