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Iminosugar C-Glycosides Work as Pharmacological Chaperones of NAGLU, a Glycosidase Involved in MPS IIIB Rare Disease*.
Zhu S, Jagadeesh Y, Tran AT, Imaeda S, Boraston A, Alonzi DS, Poveda A, Zhang Y, Désiré J, Charollais-Thoenig J, Demotz S, Kato A, Butters TD, Jiménez-Barbero J, Sollogoub M, Blériot Y. Zhu S, et al. Among authors: charollais thoenig j. Chemistry. 2021 Aug 5;27(44):11291-11297. doi: 10.1002/chem.202101408. Epub 2021 Jul 2. Chemistry. 2021. PMID: 34106504
4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations.
Front S, Almeida S, Zoete V, Charollais-Thoenig J, Gallienne E, Marmy C, Pilloud V, Marti R, Wood T, Martin OR, Demotz S. Front S, et al. Among authors: charollais thoenig j. Bioorg Med Chem. 2018 Nov 1;26(20):5462-5469. doi: 10.1016/j.bmc.2018.09.023. Epub 2018 Sep 21. Bioorg Med Chem. 2018. PMID: 30270003
(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.
Front S, Biela-Banaś A, Burda P, Ballhausen D, Higaki K, Caciotti A, Morrone A, Charollais-Thoenig J, Gallienne E, Demotz S, Martin OR. Front S, et al. Among authors: charollais thoenig j. Eur J Med Chem. 2017 Jan 27;126:160-170. doi: 10.1016/j.ejmech.2016.09.095. Epub 2016 Sep 29. Eur J Med Chem. 2017. PMID: 27750150