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    • 66. 发明专利
    • Modulators of cystic fibrosis transmembrane conductance regulator
    • NZ581259A
    • 2012-07-27
    • NZ58125908
    • 2008-05-09
    • VERTEX PHARMA
    • HADIDA RUAH SARAGROOTENHUIS PETER D
    • C07D401/04A61K31/444A61P11/00A61P37/00C07D405/14
    • Disclosed herein are pyridyl-carboxamide compounds of formula I, wherein the substituents are as defined within the specification, processes for their preparation, compositions comprising said compounds and uses thereof. Said compounds are useful as modulators of Cystic Fibrosis Transmembrane Conductance Regulators (CFTR) and are therefore useful in the treatment of cystic fibrosis, hereditary emphysema, hereditary hemochromatosis, coagulation-fibrinolysis deficiencies, protein C deficiency, Type 1 hereditary angioedema, lipid processing deficiencies, familial hypercholesterolemia, Type 1 chylomicronemia, abetalipoproteinemia, lysosomal storage diseases, I-cell disease/pseudo-Hurler, mucopolysaccharidoses, Sandhof/Tay-Sachs, Crigler-Najjar type II, polyendocrinopathy/hyperinsulemia, Diabetes mellitus, Laron dwarfism, myeloperoxidase deficiency, primary hypoparathyroidism, melanoma, glycanosis CDG type 1, congenital hyperthyroidism, osteogenesis imperfecta, hereditary hypofibrinogenemia, ACT deficiency, Diabetes insipidus (DI), neurophyseal DI, neprogenic DI, Charcot-Marie Tooth syndrome, Perlizaeus-Merzbacher disease, neurodegenerative diseases Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, progressive supranuclear plasy, Pick's disease, several polyglutamine neurological disorders, Huntington, spinocerebullar ataxia type I, spinal and bulbar muscular atrophy, dentatorubal pallidoluysian, and myotonic dystrophy, as well as spongiform encephalopathies, hereditary Creutzfeldt-Jakob disease (due to prion protein processing defect), Fabry disease, Straussler-Scheinker syndrome, COPD, dry-eye disease, or Sjogren's disease.