Modulators of atp-binding cassette transporters

    公开(公告)号:NZ719598A

    公开(公告)日:2017-03-31

    申请号:NZ71959808

    申请日:2008-11-12

    Applicant: VERTEX PHARMA

    Abstract: Disclosed herein is a use of an indol-cyclopropanecarboxamide derivative represented by structural formula shown herein in the manufacture of a medicament for treating a condition, disease, or disorder in a patient implicated by ABC transporter activity. Said compound is a modulator of ATP-Binding Cassette (’ABC’) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and is also suitable for treating a condition, disease, or disorder selected from cystic fibrosis, hereditary emphysema, hereditary hemochromatosis, coagulation-fibrinolysis deficiencies, lipid processing deficiencies, lysosomal storage diseases, neurodegenerative diseases, polyglutamine neurological disorders, spongiform encephalopathies, chronic obstructive pulmonary disease (COPD), dry-eye disease, and Sjögren’s disease.

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