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公开(公告)号：US08349319B2
公开(公告)日：2013-01-08
申请号：US12870790
申请日：2010-08-28
IPC分类号： A61K38/46
CPC分类号： A61K38/465 , C12Y301/04012
摘要： The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.
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2.发明申请DOSE ESCALATION ENZYME REPLACEMENT THERAPY FOR TREATING ACID SPHINGOMYELINASE DEFICIENCY 有权用于治疗酸性磷酸酶缺乏症的剂量自动化酶替代疗法公开(公告)号：US20110052559A1
公开(公告)日：2011-03-03
申请号：US12870790
申请日：2010-08-28
CPC分类号： A61K38/465 , C12Y301/04012
摘要： The invention relates to dose escalation enzyme replacement therapy using acid sphingomyelinase (ASM) for the treatment of human subjects having acid sphingomyelinase deficiency (ASMD), and, in particular, patients with non-neurological manifestations of Niemann-Pick Disease (NPD), and in certain embodiments, NPD type B.
摘要翻译： 本发明涉及使用酸性鞘磷脂酶（ASM）用于治疗具有酸性鞘磷脂酶缺乏症（ASMD）的人类受试者，特别是尼曼 - 皮克病（NPD）的非神经病学表现的患者的剂量递增酶替代疗法，以及 在某些实施方案中，NPD型B。
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公开(公告)号：US5686240A
公开(公告)日：1997-11-11
申请号：US250740
申请日：1994-05-27
IPC分类号： G01M1/38 , A01K67/027 , A61K38/00 , C12N9/16 , C12N15/55 , C12N15/85 , C12Q1/68 , G01M1/20 , G01M1/32 , C07H21/02 , C07H21/04 , C12P19/34
CPC分类号： C12Q1/6883 , A01K67/0278 , C12N15/8509 , C12N9/16 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/0306 , A61K38/00 , C12Q2600/156 , C12Q2600/158 , Y10S435/81
摘要： The present invention relates to the acid sphingomyelinase gene and to methods of diagnosing Niemann-Pick disease. It is based, at least in part, on the cloning and expression of the full-length cDNA encoding acid sphingomyelinase, the cloning and characterization of the genomic structure of the acid sphingomyelinase gene, and on the discovery of frequent mutations in the acid sphingomyelinase gene of Ashkenazi Jewish Niemann-Pick disease patients.
摘要翻译： 本发明涉及酸性鞘磷脂酶基因和诊断尼曼 - 皮克病的方法。 它至少部分地基于编码酸性鞘磷脂酶的全长cDNA的克隆和表达，酸性鞘磷脂酶基因的基因组结构的克隆和表征以及酸性鞘磷脂酶基因中频繁突变的发现 的Ashkenazi犹太尼曼 - 皮克病患者。
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公开(公告)号：US5773278A
公开(公告)日：1998-06-30
申请号：US695472
申请日：1991-05-03
IPC分类号： G01M1/38 , A01K67/027 , A61K38/00 , C12N9/16 , C12N15/55 , C12N15/85 , C12Q1/68 , G01M1/20 , G01M1/32 , C12N5/06 , C07H21/04
CPC分类号： C12Q1/6883 , A01K67/0278 , C12N15/8509 , C12N9/16 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/0306 , A61K38/00 , C12Q2600/156 , C12Q2600/158 , Y10S435/81
摘要： The present invention relates to the acid sphingomyelinase gene and to methods of diagnosing Niemann-Pick disease. It is based, at least in part, on the cloning and expression of the full-length cDNA encoding acid sphingomyelinase and on the discovery of mutations in the acid sphingomyelinase gene of Ashkenazi Jewish Niemann-Pick disease patients.
摘要翻译： 本发明涉及酸性鞘磷脂酶基因和诊断尼曼 - 皮克病的方法。 它至少部分地基于编码酸性鞘磷脂酶的全长cDNA的克隆和表达，以及在Ashkenazi犹太尼曼 - 皮克病患者的酸性鞘磷脂酶基因中发现突变。
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公开(公告)号：US07750050B2
公开(公告)日：2010-07-06
申请号：US10998270
申请日：2004-11-24
CPC分类号： A61K38/465 , A61K31/74 , A61K45/06 , C12Y301/04012 , A61K2300/00
摘要： The present invention provides a method for treating individuals affected with the acid sphingomyelinase-deficient forms of Niemann-Pick disease (i.e., Type A or Type B Niemann-Pick) by administering small molecules as specific molecular “chaperones” for the deficient acid sphingomyelinase (ASM) enzyme associated with the disease. The molecules are ceramide, sphingomyelin, or phosphonucleotide analogues.
摘要翻译： 本发明提供了一种通过给予缺陷型酸性鞘磷脂酶作为特异性分子“伴侣”的小分子来治疗受到尼曼 - 皮克病酸性鞘磷脂酶缺陷形式（即A型或B型尼曼选择）影响的个体的方法（ ASM）与疾病相关的酶。 分子是神经酰胺，鞘磷脂或磷酸核苷酸类似物。
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6.发明授权Acid sphingomyelinase protein and methods of treating type B Niemann-Pick disease 失效酸性鞘磷脂酶蛋白和治疗B型尼曼 - 皮克病的方法公开(公告)号：US06541218B1
公开(公告)日：2003-04-01
申请号：US09106375
申请日：1998-06-29
IPC分类号： C07H2102
CPC分类号： C12Q1/6883 , A01K67/0278 , A01K2207/15 , A01K2217/00 , A01K2217/05 , A01K2227/105 , A01K2267/0306 , A61K38/00 , C12N9/16 , C12N15/8509 , C12Q2600/156 , C12Q2600/158 , Y10S435/81
摘要： The present invention relates to the acid sphingomyelinase gene and to methods of diagnosing Niemann-Pick disease. It is based, at least in part, on the cloning and expression of the full-length cDNA encoding acid sphingomyelinase and on the discovery of mutations in the acid sphingomyelinase gene of Ashkenazi Jewish Niemann-Pick disease patients.
摘要翻译： 本发明涉及酸性鞘磷脂酶基因和诊断尼曼 - 皮克病的方法。 它至少部分地基于编码酸性鞘磷脂酶的全长cDNA的克隆和表达，以及在Ashkenazi犹太尼曼 - 皮克病患者的酸性鞘磷脂酶基因中发现突变。
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7.发明申请ANTI-TNF-ALPHA THERAPY FOR THE MUCOPOLYSACCHARIDOSES AND OTHER LYSOSOMAL DISORDERS 有权抗肿瘤坏死因子-α-阿尔法治疗脑血管病和其他淋巴细胞病公开(公告)号：US20140205584A1
公开(公告)日：2014-07-24
申请号：US14128411
申请日：2012-06-20
IPC分类号： A61K38/46 , A61K45/06 , A61K31/737
CPC分类号： A61K38/465 , A61K31/737 , A61K38/46 , A61K45/06 , A61K2300/00
摘要： The present invention relates to methods of treating a subject with a lysosomal disorder, by administering an agent for enzyme replacement therapy and an agent for anti-TNF-α therapy; by administering a pentosan polysulfate therapy; or by administering a substrate reduction therapy and an anti-TNF-α therapy. The invention further relates to a method of reducing inflammatory cytokines in a subject with a lysosomal disorder that is being treated by enzyme replacement therapy, by administering an agent for anti-TNF-α therapy.
摘要翻译： 本发明涉及通过施用酶替代治疗剂和抗TNF-α治疗剂来治疗患有溶酶体病症的受试者的方法; 通过施用戊聚糖多硫酸盐治疗; 或通过施用底物还原疗法和抗TNF-α治疗。 本发明还涉及通过施用抗TNF-α治疗剂来减少患有通过酶替代疗法治疗的溶酶体病症的受试者的炎性细胞因子的方法。
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公开(公告)号：US10350277B2
公开(公告)日：2019-07-16
申请号：US14343150
申请日：2012-09-07
摘要： The present invention relates to methods of producing chondrocytes, improving the phenotype of a chondrocyte population, promoting chondrogenesis, maintaining a cell population in a differentiated state or increasing the number of cells of a population in a differentiated state, producing a population of differentiated cells, and treating certain diseases or disorders. Each of these methods involves treating a cell population with, and/or culturing a cell population in, a ceramidase.
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9.发明授权Anti-TNF-α therapy for the mucopolysaccharidoses and other lysosomal disorders 有权抗TNF-α治疗粘多糖贮积和其他溶酶体障碍公开(公告)号：US09155784B2
公开(公告)日：2015-10-13
申请号：US14128411
申请日：2012-06-20
IPC分类号： A61K31/737 , A61K38/46 , A61K45/06
CPC分类号： A61K38/465 , A61K31/737 , A61K38/46 , A61K45/06 , A61K2300/00
摘要： The present invention relates to methods of treating a subject with a lysosomal disorder, by administering an agent for enzyme replacement therapy and an agent for anti-TNF-α therapy; by administering a pentosan polysulfate therapy; or by administering a substrate reduction therapy and an anti-TNF-α therapy. The invention further relates to a method of reducing inflammatory cytokines in a subject with a lysosomal disorder that is being treated by enzyme replacement therapy, by administering an agent for anti-TNF-α therapy.
摘要翻译： 本发明涉及通过施用酶替代治疗剂和抗TNF-α治疗剂来治疗患有溶酶体病症的受试者的方法; 通过施用戊聚糖多硫酸盐治疗; 或通过施用底物还原疗法和抗TNF-α治疗。 本发明还涉及通过施用抗TNF-α治疗剂来减少患有通过酶替代疗法治疗的溶酶体病症的受试者的炎性细胞因子的方法。
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公开(公告)号：US08961962B2
公开(公告)日：2015-02-24
申请号：US11970393
申请日：2008-01-07
IPC分类号： A61K38/50 , A61P15/00 , C12Q1/37 , C12N5/075 , C12N5/00 , C12N5/073 , C12N5/0793 , C12N9/80 , G01N33/50
CPC分类号： C12N5/0609 , C12N5/0018 , C12N5/0604 , C12N5/0619 , C12N9/80 , C12N2501/48 , C12N2501/70 , C12Y305/01023 , G01N33/5058 , G01N33/5073 , G01N2333/98
摘要： The present invention relates to methods of promoting the survival of cells by treating the cells with acid ceramidase. A kit for promoting ex vivo cell survival is also disclosed, as is a method of predicting in vitro fertilization outcome of a female subject.
摘要翻译： 本发明涉及通过用酸性神经酰胺酶处理细胞来促进细胞存活的方法。 还公开了用于促进体外细胞存活的试剂盒，以及预测雌性受试者体外受精结果的方法。
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