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公开(公告)号：EP2723384B1
公开(公告)日：2018-08-29
申请号：EP12802206.8
申请日：2012-06-22
IPC分类号： A61K47/00 , A61P25/28 , G01N33/573
CPC分类号： C07D487/04 , C12Q1/34 , C12Q1/44 , G01N33/5008 , G01N33/573 , G01N2333/924 , G01N2500/10 , Y02A50/57
摘要： The present disclosure provides technologies relating to lysosomal activation. The disclosure provides several strategies for increasing level and/or activity of lysosomal enzyme, and furthermore demonstrates the surprising applicability of such strategies in the treatment and/or prophylaxis of certain proteinopathies. Among other things, the present invention provides methods and compositions for the treatment and/or prophylaxis of proteinopathies other than lysosomal storage diseases through lysosomal activation. In particular, the present disclosure provides methods and compositions for the treatment and/or prophylaxis of neurodegenerative proteinopathies, and in particular those associated with accumulation of α-synuclein. The present disclosure specifically provides methods and compositions for the treatment and/or prophylaxis of Parkinson's disease.
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公开(公告)号：EP2723384A1
公开(公告)日：2014-04-30
申请号：EP12802206.8
申请日：2012-06-22
IPC分类号： A61K47/00 , A61P25/28 , G01N33/573
CPC分类号： C07D487/04 , C12Q1/34 , C12Q1/44 , G01N33/5008 , G01N33/573 , G01N2333/924 , G01N2500/10 , Y02A50/57
摘要： The present disclosure provides technologies relating to lysosomal activation. The disclosure provides several strategies for increasing level and/or activity of lysosomal enzyme, and furthermore demonstrates the surprising applicability of such strategies in the treatment and/or prophylaxis of certain proteinopathies. Among other things, the present invention provides methods and compositions for the treatment and/or prophylaxis of proteinopathies other than lysosomal storage diseases through lysosomal activation. In particular, the present disclosure provides methods and compositions for the treatment and/or prophylaxis of neurodegenerative proteinopathies, and in particular those associated with accumulation of α-synuclein. The present disclosure specifically provides methods and compositions for the treatment and/or prophylaxis of Parkinson's disease.
摘要翻译： 本公开提供了与溶酶体活化有关的技术。 本公开内容提供了用于提高溶酶体酶的水平和/或活性的几种策略，并且进一步证实了这些策略在治疗和/或预防某些蛋白质病中的令人惊讶的适用性。 除其他之外，本发明提供了用于通过溶酶体活化治疗和/或预防溶酶体贮积病之外的蛋白病的方法和组合物。 具体而言，本公开提供了用于治疗和/或预防神经变性蛋白病的方法和组合物，特别是与α-突触核蛋白积聚有关的方法和组合物。 本公开具体提供了用于治疗和/或预防帕金森病的方法和组合物。
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