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    Heteroaryl derivatives as cftr modulators
    33.
    发明专利
    Heteroaryl derivatives as cftr modulators 未知

    公开(公告)号：NZ703814A

    公开(公告)日：2016-06-24

    申请号：NZ70381409

    申请日：2009-02-25

    Applicant: VERTEX PHARMA

    Inventor： HADIDA-RUAH SARA ,  MILLER MARK ,  ZHOU JINGLAN ,  BEAR BRIAN

    IPC: C07D239/42 ,  A61K31/435 ,  A61K31/4965 ,  A61K31/505 ,  A61P3/00 ,  A61P25/00 ,  C07D213/75 ,  C07D241/20

    Abstract: Disclosed herein are 1-(benzo[d][1,3]dioxol-5-yl)-carboxamide compounds of formula (I), wherein the substituents are as defined in the specification, processes for its preparation, compositions comprising said compound and uses thereof. These compounds are useful as modulators of ATP-Binding Cassette (ABC) transporter or fragments thereof and is therefore useful in the treatment of condition, disease or disorders selected from cystic fibrosis, hereditary emphysema, hereditary hemochromatosis, protein C deficiency, Type 1 hereditary angioedema, familial hypercholesterolemia, Type 1 chylomicronemia, abetalipoproteinemia, 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, hereditary emphysema, congenital hyperthyroidism, osteogenesis imperfecta, hereditary hypofibrinogenemia, ACT deficiency, diabetes insipidus (di), neurophyseal di, neprogenic DI, Charcot-Marie Tooth syndrome, Perlizaeus-Merzbacher disease, Alzheimer’s disease, Parkinson’s disease, amyotrophic lateral sclerosis, progressive supranuclear plasy, Pick’s disease, Huntington, spinocerebullar ataxia type I, spinal and bulbar muscular atrophy, dentatorubal pallidoluysian, myotonic dystrophy, hereditary Creutzfeldt-Jakob disease, Fabry disease, Straussler-Scheinker syndrome, COPD, dry-eye disease, and Sjögren’s disease.

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