Afegostat HCl

Description: Afegostat is an acid beta-glucosidase (GCase) active site inhibitor that acts as a pharmacological chaperone. Afegostat inhibits GCase with K(i) ~30 nM for wild-type and mutant enzymes. Afegostat was an experimental drug for the treatment of certain forms of Gaucher's disease. Target: Others. Smiles: C(O)[C@@H]1[C@@H](O)[C@H](O)CNC1.Cl. References: Front S, Almeida S, Zoete V, Charollais-Thoenig J, Gallienne E, Marmy C, Pilloud V, Marti R, Wood T, Martin OR, Demotz S. 4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to beta-galactosidase mutations: Improved synthesis and biological investigations. Bioorg Med Chem. 2018 Nov 1,26(20):5462-5469. doi: 10.1016/j.bmc.2018.09.023. Epub 2018 Sep 21. PubMed PMID: 30270003.