Article
Discovery of N-(3-Carbamoyl-5,5,7,7-tetramethyl-5,7-dihydro-4H-thieno[2,3-c]pyran-2-yl)-lH-pyrazole-5-carboxamide (GLPG1837), a Novel Potentiator Which Can Open Class III Mutant Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Channels to a High Extent.
Journal of medicinal chemistry - 22 Feb 2018
Van der Plas Steven E, Kelgtermans Hans, De Munck Tom, Martina Sébastien L X, Dropsit Sébastien, Quinton Evelyne, De Blieck Ann, Joannesse Caroline, Tomaskovic Linda, Jans Mia, Christophe Thierry, van der Aar Ellen, Borgonovi Monica, Nelles Luc, Gees Maarten, Stouten Pieter, Van Der Schueren Jan, Mammoliti Oscar, Conrath Katja, Andrews Martin
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations in the gene for the cystic fibrosis transmembrane conductance regulator (CFTR). With the discovery of Ivacaftor and Orkambi, it has been shown that CFTR function can be partially restored by administering one or more small molecules. These molecules aim at either enhancing the amount of CFTR on the cell surface (correctors) or at improving the gating function of the...
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