Fredrick Van Goor

Discovery of N-(2,4-di-tert-butyl-5-hydroxyphenyl)-4-oxo-1,4-dihydroquinoline-3-carboxamide (VX-770, ivacaftor), a potent and orally bioavailable CFTR potentiator.

Jinglan Zhou, P. Negulescu, P. Grootenhuis, 2014, Journal of medicinal chemistry.

Use of primary cultures of human bronchial epithelial cells isolated from cystic fibrosis patients for the pre-clinical testing of CFTR modulators.

F. van Goor, B. Burton, Tim Neuberger, 2011, Methods in molecular biology.

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

Jinglan Zhou, P. Negulescu, P. Grootenhuis, 2009, Proceedings of the National Academy of Sciences.

Ivacaftor potentiation of multiple CFTR channels with gating mutations.

F. van Goor, Haihui Yu, B. Burton, 2012, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

Sweat chloride as a biomarker of CFTR activity: proof of concept and ivacaftor clinical trial data.

J. Clancy, F. van Goor, F. Accurso, 2014, Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society.

Rescue of DeltaF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules.

James Rader, P. Negulescu, T. Knapp, 2006, American journal of physiology. Lung cellular and molecular physiology.

Correction of the F508del-CFTR protein processing defect in vitro by the investigational drug VX-809

P. Negulescu, P. Grootenhuis, J. Wine, 2011, Proceedings of the National Academy of Sciences.

Dependence of Pituitary Hormone Secretion on the Pattern of Spontaneus Voltage-gated Calcium Influx

Stanko S. Stojilkovic, S. Stojilkovic, F. van Goor, 2001, The Journal of Biological Chemistry.

Defining the disease liability of variants in the cystic fibrosis transmembrane conductance regulator gene

George P Patrinos, Rachel Karchin, David L. Masica, 2013, Nature Genetics.

VX-809 corrects folding defects in cystic fibrosis transmembrane conductance regulator protein through action on membrane-spanning domain 1

Hong Yu Ren, D. Cyr, F. van Goor, 2013, Molecular biology of the cell.