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Heteroaryl derivatives as cftr modulators
专利权人:
VERTEX PHARMACEUTICALS INCORPORATED
发明人:
HADIDA-RUAH SARA,MILLER MARK,ZHOU JINGLAN,BEAR BRIAN
申请号:
NZ60203009
公开号:
NZ602030A
申请日:
2009.02.25
申请国别(地区):
NZ
年份:
2014
代理人:
摘要:
Disclosed herein are 1-(benzo[d][1,3]dioxol-5-yl)-carboxamide compounds of formula I, wherein the substituents are as defined within the specification, processes for its preparation, compositions comprising said compound and uses thereof. Said 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, myleoperoxidase 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&rsquos disease, Parkinson&rsquos disease, amyotrophic lateral sclerosis, progressive supranuclear plasy, Pick&rsquos 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&rsquos disease.
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