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1. 发明申请

WO2011049737A1 NOVEL COMPOSITIONS FOR PREVENTING AND/OR TREATING LYSOSOMAL STORAGE DISORDERS 审中-公开
标题翻译：用于预防和/或治疗LYSOSOMAL存储障碍的新组合物
公开(公告)号：WO2011049737A1
公开(公告)日：2011-04-28
申请号：PCT/US2010/051458
申请日：2010-10-05
申请人： AMICUS THERAPEUTICS, INC. , BOYD, Robert , LEE, Gary , RYBCZYNSKI, Philip
发明人： BOYD, Robert , LEE, Gary , RYBCZYNSKI, Philip
IPC分类号： A01N43/40 , A61K31/445
CPC分类号： C07D211/46 , A61K31/15 , A61K31/445 , A61K38/47 , C07D211/96
摘要： The present invention provides novel compositions as well as methods for preventing and/or treating lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease.
摘要翻译：本发明提供了新的组合物以及用于预防和/或治疗溶酶体贮积症的方法。特别地，本发明提供了预防和/或治疗戈谢氏病的方法。

2. 发明申请

WO2011049736A1 NOVEL COMPOSITIONS FOR PREVENTING AND/OR TREATING DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM 审中-公开
标题翻译：用于预防和/或治疗中枢神经系统的变应性疾病的新组合物
公开(公告)号：WO2011049736A1
公开(公告)日：2011-04-28
申请号：PCT/US2010/051447
申请日：2010-10-05
申请人： AMICUS THERAPEUTICS, INC. , BOYD, Robert , LEE, Gary , RYBCZYNSKI, Philip
发明人： BOYD, Robert , LEE, Gary , RYBCZYNSKI, Philip
IPC分类号： A01N43/40 , A61K31/445
CPC分类号： C07D211/46 , A61K31/191 , A61K31/445 , A61K31/451 , A61K45/06 , C07D211/44 , C07D211/60 , C07D211/96 , A61K2300/00
摘要： The present invention provides novel compounds as well as compositions and methods using the same for preventing and/or treating degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Parkinson's disease.
摘要翻译：本发明提供了新的化合物以及使用其的组合物和方法，用于预防和/或治疗中枢神经系统的退行性疾病。特别地，本发明提供了用于预防和/或治疗帕金森病的方法。

3. 发明申请

WO2010118283A1 METHODS FOR PREVENTING AND/OR TREATING LYSOSOMAL STORAGE DISORDERS 审中-公开
标题翻译：预防和/或治疗淋巴细胞储存障碍的方法
公开(公告)号：WO2010118283A1
公开(公告)日：2010-10-14
申请号：PCT/US2010/030472
申请日：2010-04-09
申请人： AMICUS THERAPEUTICS, INC. , BOYD, Robert , LEE, Gary
发明人： BOYD, Robert , LEE, Gary
IPC分类号： A01N43/40 , A61K31/445
CPC分类号： C12Q1/34 , A61K31/445 , C07D211/36 , G01N2333/924
摘要： The present invention provides methods for preventing and/or treating lysosomal storage disorders using 5-(fluoromethyl)piperdine-3,4-diol, 5- (chloromethyl)piperdine-3,4-diol, or a pharmaceutically acceptable salt, solvate, or prodrug thereof, or any combination of two or more thereof. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease.
摘要翻译：本发明提供使用5-（氟甲基）哌啶-3,4-二醇，5-（氯甲基）哌啶-3,4-二醇或其药学上可接受的盐，溶剂合物或/或溶剂合物来预防和/或治疗溶酶体贮积障碍的方法前药，或其两种或多种的任何组合。特别地，本发明提供了预防和/或治疗戈谢氏病的方法。

4. 发明申请

WO2010118282A1 METHODS FOR PREVENTING AND/OR TREATING DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM 审中-公开
标题翻译：用于预防和/或治疗中枢神经系统的变应性疾病的方法
公开(公告)号：WO2010118282A1
公开(公告)日：2010-10-14
申请号：PCT/US2010/030470
申请日：2010-04-09
申请人： AMICUS THERAPEUTICS, INC. , BOYD, Robert , LEE, Gary
发明人： BOYD, Robert , LEE, Gary
IPC分类号： A01N43/40 , A61K31/445
CPC分类号： A61K31/445
摘要： The present invention provides methods for preventing and/or treating degenerative disorders of the central nervous system using 5- (fluoromethyl)piperdine-3,4-diol, 5-(chloromethyl)piperdine-3,4-diol, or a pharmaceutically acceptable salt, solvate, or prodrug thereof, or any combination of two or more thereof. In particular, the present invention provides methods for preventing and/or treating Parkinson's disease.
摘要翻译：本发明提供使用5-（氟甲基）哌啶-3,4-二醇，5-（氯甲基）哌啶-3,4-二醇或其药学上可接受的盐来预防和/或治疗中枢神经系统的退行性疾病的方法，溶剂合物或前药，或其两种或多种的任何组合。特别地，本发明提供了用于预防和/或治疗帕金森病的方法。

5. 发明申请

WO2011049787A1 METHOD FOR TREATING ALZHEIMER'S DISEASE USING PHARMACOLOGICAL CHAPERONES TO INCREASE THE ACTIVITY OF GANGLIOSIDASES 审中-公开
标题翻译：使用药理学治疗增加疟原虫活性的阿尔茨海默病的方法
公开(公告)号：WO2011049787A1
公开(公告)日：2011-04-28
申请号：PCT/US2010/052351
申请日：2010-10-12
申请人： AMICUS THERAPEUTICS, INC. , WUSTMAN, Brandon , VALENZANO, Kenneth , BOYD, Robert
发明人： WUSTMAN, Brandon , VALENZANO, Kenneth , BOYD, Robert
IPC分类号： A61K38/47 , A61F2/00
CPC分类号： A61K31/7008 , A61K31/426 , A61K31/429 , A61K31/445 , A61K38/1709 , G06F19/00
摘要： The present invention relates to a method for treating an individual having Alzheimer's Disease by using pharmacological chaperones to increase the activity of gangliosidase and/or sialidase enzymes involved in ganglioside catabolism.
摘要翻译：本发明涉及通过使用药理学伴侣来增加神经节苷脂分解代谢中神经节苷脂酶和/或唾液酸酶的活性来治疗患有阿尔茨海默病的个体的方法。

6. 发明申请

WO2011011181A1 PHARMACOLOGICAL CHAPERONES FOR THE TREATMENT OF ALZHEIMER'S DISEASE 审中-公开
标题翻译：用于治疗阿尔茨海默病的药理学章节
公开(公告)号：WO2011011181A1
公开(公告)日：2011-01-27
申请号：PCT/US2010/040774
申请日：2010-07-01
申请人： AMICUS THERAPEUTICS, INC. , BOYD, Robert
发明人： BOYD, Robert
IPC分类号： A61K38/00
CPC分类号： A61K38/57 , A61K31/381
摘要： The invention relates to methods of enhancing normal α -secretase expression, and to enhancing expression of an α -secretase having a mutation which affects protein folding and/or processing of the α -secretase. The invention provides a method of treating an individual having a condition in which increased expression or activity of an α -secretase in the central nervous system would be beneficial, by administering an effective amount of a pharmacologic chaperone for the α -secretase. The invention provides α-secretase inhibitors which have been identified as specific pharmacologic chaperones which enhance expression or enzymatic activity of α -secretase in the central nervous system.
摘要翻译：本发明涉及增强正常α-分泌酶表达的方法，以及增强具有影响α-分泌酶的蛋白质折叠和/或加工的突变的α-分泌酶的表达。本发明提供了一种治疗个体的方法，所述方法通过给予α-分泌酶的有效量的药物分子伴侣，其具有中枢神经系统中α-分泌酶的增加的表达或活性将是有益的状况。本发明提供已被鉴定为增强中枢神经系统中α-分泌酶的表达或酶活性的特异性药理学伴侣的分泌酶抑制剂。

7. 发明申请

WO2018200618A1 NOVEL COMPOSITIONS FOR PREVENTING AND/OR TREATING DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM AND/OR LYSOSOMAL STORAGE DISORDERS 审中-公开
公开(公告)号：WO2018200618A1
公开(公告)日：2018-11-01
申请号：PCT/US2018/029275
申请日：2018-04-25
申请人： AMICUS THERAPEUTICS, INC.
发明人： BOYD, Robert
IPC分类号： C07C215/44 , A61K31/133 , A61P25/28
摘要： The present invention provides novel compounds as well as compositions and methods using the same for preventing and/or treating degenerative disorders of the central nervous system and/or lysosomal storage disorders. In particular, the present invention provides methods for preventing and/or treating Parkinson's disease, Alzheimer's disease and/or Gaucher's disease.

8. 发明申请

WO2015123385A1 SUGAR DERIVATIVES COMPRISING SULFUR-CONTAINING MOIETIES AND METHODS OF MAKING SAME AND METHODS OF USING THE SAME FOR THE TREATMENT OF MPS IIIC 审中-公开
标题翻译：包含含硫化合物的糖衍生物及其制备方法及其使用方法用于治疗MPS IIIC
公开(公告)号：WO2015123385A1
公开(公告)日：2015-08-20
申请号：PCT/US2015/015557
申请日：2015-02-12
申请人： AMICUS THERAPEUTICS, INC.
发明人： BOYD, Robert , LEE, Gary
IPC分类号： C07D497/04 , C07D498/04 , C07D513/04 , C07D211/46 , C07D211/56 , A61K31/429 , A61K31/435 , A61K31/4365 , A61K31/437 , A61P3/00
CPC分类号： C07D211/56 , A61K31/429 , A61K31/435 , A61K31/4365 , A61K31/437 , A61K31/445 , A61K31/45 , A61K38/43 , A61K45/06 , C07D211/46 , C07D497/04 , C07D498/04 , C07D498/14 , C07D513/04 , A61K2300/00
摘要： Described herein are modified sugar, iminosugar and azasugar compounds and methods of making same. One or more of these modified compounds contain sulfates, sulfites, sulfamates and/or sulfonamides. Also described are pharmaceutical compositions/formulations comprising these compounds, as well as methods using these modified sugar compounds for the treatment of MPS IIIC (also known as Sanfilippo Type C).
摘要翻译：本文描述的是改性糖，亚氨基二糖和azasugar化合物及其制备方法。这些改性化合物中的一种或多种含有硫酸盐，亚硫酸盐，氨基磺酸盐和/或磺酰胺。还描述了包含这些化合物的药物组合物/制剂，以及使用这些改性糖化合物治疗MPS IIIC（也称为Sanfilippo C型）的方法。

9. 发明申请

WO2017106288A1 NOVEL COMPOUNDS AND METHODS FOR THE TREATMENT OF ALZHEIMER'S DISEASE AND/OR CEREBRAL AMYLOID ANGIOPATHY 审中-公开
标题翻译：用于治疗阿尔茨海默病和/或脑淀粉样血管病的新型化合物和方法
公开(公告)号：WO2017106288A1
公开(公告)日：2017-06-22
申请号：PCT/US2016/066572
申请日：2016-12-14
申请人： AMICUS THERAPEUTICS, INC.
发明人： BOYD, Robert
IPC分类号： C07D513/04 , C07D211/56 , A61K31/445 , A61P25/28
CPC分类号： C07D513/04 , C07D211/56
摘要： Described herein are novel compounds and methods for the treatment and/or prevention of cerebral amyloidoses such as Alzheimer's disease (AD) and/or cerebral amyloid angiopathy (CAA).
摘要翻译：本文描述了用于治疗和/或预防脑淀粉样变性例如阿尔茨海默氏病（AD）和/或脑淀粉样血管病（CAA）的新化合物和方法。

10. 发明申请

WO2013148103A1 NOVEL COMPOUNDS FOR PREVENTING AND/OR TREATING LYSOSOMAL STORAGE DISORDERS AND/OR DEGENERATIVE DISORDERS OF THE CENTRAL NERVOUS SYSTEM 审中-公开
标题翻译：用于预防和/或治疗中枢神经系统的LYSOSOMAL存活障碍和/或恶化病症的新型化合物
公开(公告)号：WO2013148103A1
公开(公告)日：2013-10-03
申请号：PCT/US2013/029612
申请日：2013-03-07
申请人： AMICUS THERAPEUTICS, INC.
发明人： BOYD, Robert , RYBCZYNSKI, Philip, J. , SHETH, Kamlesh
IPC分类号： C07D211/44 , A61K31/445 , A61P25/00
CPC分类号： H01L27/10855 , A61K31/445 , A61K45/06 , C07D211/44 , H01L21/31111 , H01L21/32139 , H01L27/10814 , H01L27/10852 , H01L27/10873 , H01L27/10894 , H01L28/90
摘要： Described are novel salts of the compound (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol, as well as methods of using the same for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system. In particular, the present invention provides methods for preventing and/or treating Gaucher's disease and/or Parkinson's disease.
摘要翻译：描述了化合物（3R，4R，5S）-5-（二氟甲基）哌啶-3,4-二醇的新型盐，以及其用于预防和/或治疗溶酶体贮积障碍和/或退行性疾病的方法的中枢神经系统。特别地，本发明提供了预防和/或治疗戈谢氏病和/或帕金森病的方法。

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