Cystic fibrosis is a genetic condition caused by mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR). CFTR is complex protein found on the surface membrane of cells in a wide variety of tissues where it functions as a regulated chloride ion channel. In the lung CFTR is found on the apical membrane of the cells lining the airways.
IOWH-032
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Ivacaftor (benzenesulfonate)
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Ivacaftor hydrate
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Nesolicaftor
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PPQ-102
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PTC124
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VX-661
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VX-770
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VX-809
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