Modulators of atp-binding cassette-transporters
AssigneeVertex Pharmaceuticals Incorporated
InventorsSara S. Hadida Ruah, Mark Miller, Jinglan Zhou, Brian Bear, Peter Grootenhuis
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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.
Claims8
1-45. (canceled)
A method of treating or lessening the severity of a disease in a patient, wherein the disease is cystic fibrosis comprising the step of administering to the patient an effective amount of a compound
- 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.
47-49. (canceled)
A method of treating or lessening the severity of 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:
- or a pharmaceutically acceptable salt thereof;
- and
- ii) a pharmaceutically acceptable carrier.
Dependencies are inferred from the claim text.