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|Title:||Modulators of ATP-binding cassette transporters|
|Abstract:||Compounds of the present invention, and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette ("ABC") transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator ("CFTR"). The present invention also relates to methods of treating ABC transporter mediated diseases using compounds of the present invention.|
|Inventor(s):||Hadida Ruah; Sara S. (La Jolla, CA), Hamilton; Matthew (Hackettstown, NJ), Miller; Mark (San Diego, CA), Grootenhuis; Peter D. J. (San Diego, CA), Bear; Brian (Carlsbad, CA), McCartney; Jason (Cardiff by the Sea, CA), Zhou; Jinglan (San Diego, CA)|
|Assignee:||Vertex Pharmaceuticals Incorporated (Boston, MA)|
1. A method of treating cystic fibrosis in a patient, wherein the patient has a defective gene that causes a deletion of phenylalanine at position 508 of the cystic fibrosis
transmembrane conductance regulator amino acid sequence, said method comprising the step of administering to the patient an effective amount of the compound ##STR00524## or a pharmaceutically acceptable salt thereof.
2. The method of claim 1, wherein the patient has two copies of the defective gene.
3. A method of treating cystic fibrosis in a patient, wherein the patient has a defective gene that causes a deletion of phenylalanine at position 508 of the cystic fibrosis transmembrane conductance regulator amino acid sequence, said method comprising the step of administering to said patient an effective amount of a composition comprising: i) the compound: ##STR00525## or a pharmaceutically acceptable salt thereof; and ii) a pharmaceutically acceptable carrier.
4. The method of claim 3, wherein the patient has two copies of the defective gene.
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