|Title:||Modulators of ATP-binding cassette transporters|
|Abstract:||The present invention relates to modulators of ATP-Binding Cassette ("ABC") transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof, and methods therewith. The present invention also relates to methods of treating ABC transporter mediated diseases using such modulators.|
|Inventor(s):||Ruah; Sara Hadida (La Jolla, CA), Hazlewood; Anna (San Diego, CA), Grootenhuis; Peter D. J. (San Diego, CA), Van Goor; Fredrick (San Diego, CA), Singh; Ashvani (San Diego, CA), Zhou; Jinglan (San Diego, CA), McCartney; Jason (Cardiff-by-the-Sea, CA)|
|Assignee:||Vertex Pharmaceutical Incorporated (Cambridge, MA)|
1. A method of treating or lessening the severity of cystic fibrosis in a patient, said method comprising the step of administering to said patient an effective amount of
N-(5-hydroxy-2,4-ditert-butyl-phenyl)-4-oxo-1H-quinoline-3-carboxamide, which has the chemical structure: ##STR00242##
2. The method of claim 1, wherein the patient possesses a cystic fibrosis transmembrane receptor with a G551D mutation.
3. The method of claim 2, wherein the patient is homozygous for G551D mutation.
4. The method of claim 2, wherein the patient is heterozygous for G551D mutation.