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|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):||Van Goor; Fredrick (San Diego, CA)|
|Assignee:||Vertex Pharmaceutical Incorporated (Cambridge, MA)|
1. A method of treating or lessening the severity of cystic fibrosis in a patient, wherein said patient possess a cystic fibrosis transmembrane receptor (CFTR) with a R117H
mutation, 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.
2. The method of claim 1, wherein the patient is heterozygous for R117H mutation.
3. The method of claim 1, wherein the patient is homozygous for R117H mutation.