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Isoquinoline modulators of ATP-Binding Cassette transporters
专利权人:
VERTEX PHARMACEUTICALS INCORPORATED
发明人:
HADIDA RUAH, SARA,MILLER, MARK,ZHOU, JINGLAN,BEAR, BRIAN,GROOTENHUIS, PETER D
申请号:
NZ59894108
公开号:
NZ598941A
申请日:
2008.11.14
申请国别(地区):
NZ
年份:
2013
代理人:
摘要:
ABSTRACT - 598941 The disclosure relates to a substituted isoquinoline derivative of the formula (I) and pharmaceutically acceptable salt thereof, wherein the variables are as defined in the specification. These isoquinoline derivatives are useful as modulators of ATPBinding Cassette ("ABC") transporter activity, including Cystic Fibrosis Transmembrane Conductance Regulator ("CFTR"). Said substituted isoquinoline compound may be suitable for treating ABC transporter mediated diseases selected from cystic fibrosis, asthma, smoke induced COPD, chronic bronchitis, rhinosinusitis, constipation, pancreatitis, pancreatic insufficiency, male infertility caused by congenital bilateral absence of the vas deferens, mild pulmonary disease, idiopathic pancreatitis, allergic bronchopulmonary aspergillosis, liver disease, hereditary emphysema, hereditary hemochromatosis, coagulation-fibrinolysis deficiencies, lipid processing deficiencies, lysosomal storage diseases, 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, neurophyseal DI, neprogenic DI, Charcot-Marie Tooth syndrome, Perlizaeus-Merzbacher disease, neurodegenerative diseases several polyglutamine neurological disorders as well as spongiform encephalopathies, COPD, dry-eye disease and Sjogrens disease.
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