公开(公告)号：US20120322787A1

公开(公告)日：2012-12-20

申请号：US13595349

申请日：2012-08-27

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/4025 ,  A61P3/10 ,  A61P13/12 ,  C07D409/14 ,  A61K31/4439 ,  A61K31/397 ,  A61K31/422 ,  C07D417/14 ,  A61K31/428 ,  A61K31/4245 ,  A61K31/444 ,  A61K31/5377 ,  C07D405/10 ,  C07D413/14

CPC分类号： C07D417/14 ,  C07D295/125 ,  C07D319/18 ,  C07D405/06 ,  C07D405/12 ,  C07D405/14 ,  C07D407/12 ,  C07D409/12 ,  C07D409/14 ,  C07D411/12 ,  C07D413/12 ,  C07D413/14

摘要： A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-α, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 治疗有需要的受试者的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 受试者患有2型糖尿病; 肾脏肥大或与糖尿病肾病相关的增生; 泰撒 戈谢的 或法布里氏病。 分别在有需要的受试者中降低血浆TNF-α，降低血糖水平，降低糖化血红蛋白水平，抑制葡萄糖神经酰胺合成酶和降低糖鞘醇浓度的方法包括向受试者施用治疗有效量的由结构式（ I）或其药学上可接受的盐。

公开(公告)号：US20110184021A1

公开(公告)日：2011-07-28

申请号：US13122135

申请日：2009-10-02

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozafiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozafiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/453 ,  A61K31/40 ,  A61K31/4025 ,  A61K31/4439 ,  A61K31/422 ,  A61K31/428 ,  A61P13/12

CPC分类号： A61K31/4025 ,  C07C271/12 ,  C07D207/12 ,  C07D317/60 ,  C07D319/18 ,  C07D319/20 ,  C07D333/16 ,  C07D407/06 ,  C07D407/12 ,  C07D409/04 ,  C07D413/04 ,  C07D417/12

摘要： A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 用于治疗多囊肾病的化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 分别在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。

公开(公告)号：US20120322786A1

公开(公告)日：2012-12-20

申请号：US13595251

申请日：2012-08-27

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozafiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozafiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/4025 ,  A61K31/4439 ,  A61K31/397 ,  A61K31/428 ,  A61K31/5377 ,  A61K31/40 ,  A61K31/422

CPC分类号： A61K31/4025 ,  C07C271/12 ,  C07D207/12 ,  C07D317/60 ,  C07D319/18 ,  C07D319/20 ,  C07D333/16 ,  C07D407/06 ,  C07D407/12 ,  C07D409/04 ,  C07D413/04 ,  C07D417/12

摘要： A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 用于治疗多囊肾病的化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 分别在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。

公开(公告)号：US08309593B2

公开(公告)日：2012-11-13

申请号：US13122135

申请日：2009-10-02

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozafiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozafiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/4025 ,  A61K31/40

CPC分类号： A61K31/4025 ,  C07C271/12 ,  C07D207/12 ,  C07D317/60 ,  C07D319/18 ,  C07D319/20 ,  C07D333/16 ,  C07D407/06 ,  C07D407/12 ,  C07D409/04 ,  C07D413/04 ,  C07D417/12

摘要： A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 用于治疗多囊肾病的化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 分别在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。

公开(公告)号：US09272996B2

公开(公告)日：2016-03-01

申请号：US13595251

申请日：2012-08-27

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozefiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Thomas H. Jozefiak ,  Frederic Vinick ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/397 ,  A61K31/5377 ,  A61K31/44 ,  A61K31/325 ,  A61K31/42 ,  A61K31/40 ,  C07D207/12 ,  C07D271/12 ,  C07D317/60 ,  C07D319/18 ,  C07D333/16 ,  C07D407/12 ,  C07D409/04 ,  C07D413/04 ,  C07D417/12 ,  C07C271/12 ,  C07D319/20 ,  C07D407/06

CPC分类号： A61K31/4025 ,  C07C271/12 ,  C07D207/12 ,  C07D317/60 ,  C07D319/18 ,  C07D319/20 ,  C07D333/16 ,  C07D407/06 ,  C07D407/12 ,  C07D409/04 ,  C07D413/04 ,  C07D417/12

摘要： A compound for use in treating polycystic kidney disease is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating polycystic kidney disease in a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. Methods of treating in polycystic kidney disease in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 用于治疗多囊肾病的化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 分别在有需要的受试者中治疗多囊肾病的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。

公开(公告)号：US08304447B2

公开(公告)日：2012-11-06

申请号：US12601871

申请日：2008-05-30

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/40 ,  A61K31/4025 ,  C07D207/04 ,  C07D405/02

CPC分类号： C07D417/14 ,  C07D295/125 ,  C07D319/18 ,  C07D405/06 ,  C07D405/12 ,  C07D405/14 ,  C07D407/12 ,  C07D409/12 ,  C07D409/14 ,  C07D411/12 ,  C07D413/12 ,  C07D413/14

摘要： A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-α, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 治疗有需要的受试者的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 受试者患有2型糖尿病; 肾脏肥大或与糖尿病肾病相关的增生; 泰撒 戈谢的 或法布里氏病。 分别在有需要的受试者中降低血浆TNF-α，降低血糖水平，降低糖化血红蛋白水平，抑制葡萄糖神经酰胺合成酶和降低糖鞘醇浓度的方法包括向受试者施用治疗有效量的由结构式（ I）或其药学上可接受的盐。

公开(公告)号：US08940776B2

公开(公告)日：2015-01-27

申请号：US13595349

申请日：2012-08-27

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/42 ,  A61K31/4245 ,  A61K31/427 ,  A61K31/428 ,  A61K31/4025 ,  A61K31/40 ,  C07D277/20 ,  C07D277/62 ,  C07D261/06 ,  C07D405/02 ,  C07D271/06 ,  C07D207/04 ,  C07D295/125 ,  C07D319/18 ,  C07D405/06 ,  C07D405/12 ,  C07D407/12 ,  C07D409/12 ,  C07D409/14 ,  C07D411/12 ,  C07D413/12 ,  C07D413/14

CPC分类号： C07D417/14 ,  C07D295/125 ,  C07D319/18 ,  C07D405/06 ,  C07D405/12 ,  C07D405/14 ,  C07D407/12 ,  C07D409/12 ,  C07D409/14 ,  C07D411/12 ,  C07D413/12 ,  C07D413/14

摘要： A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-α, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 化合物由结构式（I）表示：或其药学上可接受的盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 治疗有需要的受试者的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 受试者患有2型糖尿病; 肾脏肥大或与糖尿病肾病相关的增生; 泰撒 戈谢的 或法布里氏病。 分别在有需要的受试者中降低血浆TNF-α，降低血糖水平，降低糖化血红蛋白水平，抑制葡萄糖神经酰胺合成酶和降低糖鞘醇浓度的方法包括向受试者施用治疗有效量的由结构式（ I）或其药学上可接受的盐。

公开(公告)号：US20100256216A1

公开(公告)日：2010-10-07

申请号：US12601871

申请日：2008-05-30

申请人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

发明人： Craig Siegel ,  Cecilia M. Bastos ,  David J. Harris ,  Angeles Dios ,  Edward Lee ,  Richard Silva ,  Lisa M. Cuff ,  Mikaela Levine ,  Cassandra A. Celatka ,  Frederic Vinick ,  Thomas H. Jozefiak ,  Yibin Xiang ,  John Kane ,  Junkai Liao

IPC分类号： A61K31/4025 ,  C07D405/06 ,  C07D207/00 ,  A61K31/4015

CPC分类号： C07D417/14 ,  C07D295/125 ,  C07D319/18 ,  C07D405/06 ,  C07D405/12 ,  C07D405/14 ,  C07D407/12 ,  C07D409/12 ,  C07D409/14 ,  C07D411/12 ,  C07D413/12 ,  C07D413/14

摘要： A compound is represented by Structural Formula (I): or a pharmaceutically acceptable salt thereof. A pharmaceutical composition comprises a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. A method of treating a subject in need thereof comprises administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof. The subject has type 2 diabetes; renal hypertrophy or hyperplasia associated with diabetic nephropathy; Tay-Sachs; Gaucher's; or Fabry's disease. Methods of decreasing plasma TNF-α, lowering blood glucose levels, decreasing glycated hemoglobin levels, inhibiting glucosylceramide synthase, and lowering glycosphingolipid concentrations in a subject in need thereof respectively comprise administering to the subject a therapeutically effective amount of a compound represented by Structural Formula (I) or a pharmaceutically acceptable salt thereof.

摘要翻译： 化合物由结构式（I）表示：或其可药用盐。 药物组合物包含由结构式（I）表示的化合物或其药学上可接受的盐。 治疗有需要的受试者的方法包括向受试者施用治疗有效量的由结构式（I）表示的化合物或其药学上可接受的盐。 受试者患有2型糖尿病; 肾脏肥大或与糖尿病肾病相关的增生; 泰撒 戈谢的 或法布里氏病。 分别在有需要的受试者中降低血浆TNF-α，降低血糖水平，降低糖化血红蛋白水平，抑制葡萄糖神经酰胺合成酶和降低糖鞘醇浓度的方法包括向受试者施用治疗有效量的由结构式（ I）或其药学上可接受的盐。