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公开(公告)号：EP2027137B1
公开(公告)日：2015-02-18
申请号：EP06772888.1
申请日：2006-06-08
IPC分类号： C07H5/04 , C07D211/40 , C07D211/46
CPC分类号： C07H5/04 , C07D211/40 , C07D211/46
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52.发明公开METHOD FOR TREATING ALZHEIMER'S DISEASE USING PHARMACOLOGICAL CHAPERONES TO INCREASE THE ACTIVITY OF GANGLIOSIDASES 有权阿尔茨海默氏病药理伴侣的治疗方法来增加GANGLIOSIDASEN的活度
公开(公告)号：EP2490712A4
公开(公告)日：2013-06-12
申请号：EP10825421
申请日：2010-10-12
发明人： WUSTMAN BRANDON , VALENZANO KENNETH , BOYD ROBERT
CPC分类号： A61K31/7008 , A61K31/426 , A61K31/429 , A61K31/445 , A61K38/1709
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.
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53.发明公开
公开(公告)号：EP2490533A1
公开(公告)日：2012-08-29
申请号：EP10825397.2
申请日：2010-10-05
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.
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54.发明公开
公开(公告)号：EP2260107A4
公开(公告)日：2011-03-23
申请号：EP09720341
申请日：2009-03-12
发明人： WUSTMAN BRANDON , DO HUNG V
CPC分类号： G01N33/6893 , G01N2333/928 , G01N2800/042 , G01N2800/52
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55.发明公开ASSAYS FOR DIAGNOSING AND EVALUATING TREATMENT OPTIONS FOR FABRY DISEASE 有权测试ZUR DIAGNOSE UNDERTEILUNG VON BEHANDLUNGSOPTIONENFÜRMORBUS FABRY
公开(公告)号：EP2024745A2
公开(公告)日：2009-02-18
申请号：EP07797506.8
申请日：2007-05-16
发明人： KANESKI, Christine , SHIN, Sang-hoon , MURRAY, Gary , LUDWIG, Karin , LOCKHART, David , CASTELLI, Jeff
IPC分类号： G01N33/53 , G01N33/567 , G01N33/573
CPC分类号： A61K31/445 , A61K9/0053 , A61K9/48 , A61K31/45 , A61K45/06 , C07D211/46 , G01N33/5094 , G01N2333/924 , G01N2333/94 , G01N2800/04 , G01N2800/52 , A61K2300/00
摘要： Provided are in vitro and in vivo methods for determining whether a patient with Fabry disease will respond to treatment with a specific pharmacological chaperone.
摘要翻译： DGJ用于治疗法布里病，其特征在于以预定的频率将DGJ以规定量施用于法布里病患者。
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公开(公告)号：EP1682504A2
公开(公告)日：2006-07-26
申请号：EP04810772.6
申请日：2004-11-12
发明人： FAN, Jian-Qiang , ZHU, Xiaoxiang , SHETH, Kamlesh
IPC分类号： C07D211/40 , A61K31/445
CPC分类号： C07D487/04
摘要： The present invention provides novel hydroxy piperidine (HP) derivatives having (i) a positive charge at the position corresponding to the anomeric position of a pyranose ring; (ii) a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and (iii) a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. The linker can be absent if the lipophilic moiety corresponds to a hydrocarbon chain with a linear length of 6 or more carbons. The present invention further provides a method for treating individuals having Gaucher disease by administering the novel HP derivative as 'chaperones' for the mutant glucocerebrosidase associated with the disease.
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57.发明公开GLUCOIMIDAZOLE AND POLYHYDROXYCYCLOHEXENYL AMINE DERIVATIVES TO TREAT GAUCHER DISEASE 审中-公开GLUCOIMIDAZOL和POLYHYDROXYCYCLOHEXENYL胺衍生物的戈谢病的治疗
公开(公告)号：EP1682134A2
公开(公告)日：2006-07-26
申请号：EP04810771.8
申请日：2004-11-12
发明人： FAN, Jian-Qiang , ZHU, Xiaoxiang , SHETH, Kamlesh
IPC分类号： A61K31/437 , C07D471/04 , A61P43/00
CPC分类号： C07D487/04
摘要： The present invention provides glucoimidazole (GIZ) and polyhydroxycyclohexenyl amine (PHCA) derivatives, methods of making them, and methods of use where the GIZ and PHCA derivatives have a short, flexible linker emanating from the corresponding position of the ring oxygen in a pyranose; and a lipophilic moiety connected to the linker and pharmaceutically acceptable salts thereof. More particularly, the present invention further provides a method for treating individuals having Gaucher disease by administering the novel GIZ or PHCA derivatives as “chaperones” for the mutant glucocerebrosidase associated with the disease.
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公开(公告)号：EP4389213A3
公开(公告)日：2024-08-21
申请号：EP24168026.3
申请日：2017-07-19
发明人： Castelli, Jeff
IPC分类号： A61K31/445 , A61P9/00 , A61P3/00 , A61P13/12
CPC分类号： A61K31/445 , A61K31/45 , A61K31/7008 , A61P13/12 , A61P3/00 , A61P39/02 , A61P43/00 , A61P9/00
摘要： Provided are dosing regimens for the treatment of Fabry disease in a patient. Certain methods relate to the treatment of ERT-experienced or ERT-naïve Fabry patients. Certain methods comprise administering to the patient about 123 mg free base equivalent of migalastat for improving left ventricular mass and/or improving podocyte globotriaosylceramide.
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59.发明授权
公开(公告)号：EP3840753B1
公开(公告)日：2024-07-24
申请号：EP19703514.0
申请日：2019-01-16
IPC分类号： A61K31/445 , A61P3/00
CPC分类号： A61K31/445 , A61P3/00
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公开(公告)号：EP3749308B1
公开(公告)日：2023-10-25
申请号：EP19706182.3
申请日：2019-02-06
发明人： BARTH, Jay , BENJAMIN, Elfrida
IPC分类号： A61K31/445 , A61P13/12
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