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1. 发明申请

WO02065989A3 AMINOGLYCOSIDE TREATMENT FOR LYSOSOMAL STORAGE DISEASES 审中-公开
标题翻译：氨基糖苷类药物治疗溶酶体贮积症
公开(公告)号：WO02065989A3
公开(公告)日：2002-12-12
申请号：PCT/US0204842
申请日：2002-02-20
申请人： UAB RESEARCH FOUNDATION , BEDWELL DAVID M , KEELING KIM M
发明人： BEDWELL DAVID M , KEELING KIM M
IPC分类号： C07H15/224 , A61K31/7008 , A61K31/7036 , A61K31/704 , A61K35/76 , A61K38/00 , A61K38/46 , A61K38/47 , A61K38/48 , A61K48/00 , A61P3/00 , A61P31/00 , A61P43/00 , C07H15/236 , A61K31/70
CPC分类号： A61K38/4813 , A61K31/7036 , A61K31/704 , A61K38/47 , A61K2300/00
摘要： The present invention provides a method of treating lysomal storage diseases such as Hurler syndrome and Batten disease in individuals in need of such treatment, comprising the step of administering to said individuals a therapeutically effective dose of an aminoglycoside. In addition, this method may further comprise treating the individual with enzyme replacement therapy. Furthermore, the present invention rovides method of pharmaceutically suppressing premature stop mutations in an indidual with these lysosomal storage diseases, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside.
摘要翻译：本发明提供了治疗需要这种治疗的个体中的溶酶体贮积病例如赫勒综合征和巴顿病的方法，包括给所述个体施用治疗有效剂量的氨基糖苷的步骤。另外，该方法可以进一步包括用酶替代疗法治疗个体。此外，本发明提供了药物抑制具有这些溶酶体贮积病的个体中的过早停止突变的方法，包括给予所述个体药理学有效剂量的氨基糖苷的步骤。

2. 发明申请

WO2002065989A2 AMINOGLYCOSIDE TREATMENT FOR LYSOSOMAL STORAGE DISEASES 审中-公开
标题翻译：用于胶原质储存疾病的氨基酰胺治疗
公开(公告)号：WO2002065989A2
公开(公告)日：2002-08-29
申请号：PCT/US2002/004842
申请日：2002-02-20
申请人： UAB RESEARCH FOUNDATION , BEDWELL, David, M. , KEELING, Kim, M.
发明人： BEDWELL, David, M. , KEELING, Kim, M.
IPC分类号： A61K
CPC分类号： A61K38/4813 , A61K31/7036 , A61K31/704 , A61K38/47 , A61K2300/00
摘要： The present invention provides a method of treating lysomal storage diseases such as Hurler syndrome and Batten disease in individuals in need of such treatment, comprising the step of administering to said individuals a therapeutically effective dose of an aminoglycoside. In addition, this method may further comprise treating the individual with enzyme replacement therapy. Furthermore, the present invention rovides method of pharmaceutically suppressing premature stop mutations in an indidual with these lysosomal storage diseases, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside.
摘要翻译：本发明提供了在需要这种治疗的个体中治疗溶酶体存储疾病如Hurler综合征和Batten病的方法，包括向所述个体施用治疗有效剂量的氨基糖苷的步骤。此外，该方法还可以包括用酶替代疗法治疗个体。此外，本发明涉及药物上抑制与这些溶酶体贮积疾病相关的过早停止突变的方法，包括向所述个体施用药理学有效剂量的氨基糖苷类的步骤。

3. 发明申请

WO2008067491A3 MATERIALS AND METHODS FOR TREATING DISEASES CAUSED BY GENETIC DISORDERS USING AMINOGLYCOSIDES AND DERIVATIVES THEREOF WHICH EXHIBIT LOW NEPHROTOXICITY 审中-公开
公开(公告)号：WO2008067491A3
公开(公告)日：2008-06-05
申请号：PCT/US2007/085974
申请日：2007-11-29
申请人： INDIANA UNIVERSITY RESEARCH AND TECHNOLOGY CORPORATION , MOLITORIS, Bruce, A. , BEDWELL, David, M. , SANDOVAL, Ruben, M.
发明人： MOLITORIS, Bruce, A. , BEDWELL, David, M. , SANDOVAL, Ruben, M.
IPC分类号： A01N43/04 , A61K31/70
摘要： Various aspects related to the preparation of congeners of the aminoglycosides gentamicin such as the congener C2 and using this compound or derivatives thereof and pharmaceutically active salts to treat diseases that involve genetic mutations which introduce a missense or premature stop codon into a gene. Still other aspects include treating human or animal patients with the gentamicin congener C2 and derivatives and pharmaceutical salt thereof to overcome, or to at least mitigate, the symptoms of disease and disorders such as some forms of Becker's or Duchenne muscular dystrophy, Hurler's Syndrome and Cystic Fibrosis that have as their etiology the presence of a premature stop codon in a gene whose proper expression is necessary for good health.

4. 发明申请

WO2008067491A2 MATERIALS AND METHODS FOR TREATING DISEASES CAUSED BY GENETIC DISORDERS USING AMINOGLYCOSIDES AND DERIVATIVES THEREOF WHICH EXHIBIT LOW NEPHROTOXICITY 审中-公开
标题翻译：使用氨基糖苷类化合物治疗疾病引起的疾病的材料和方法及其具有低耐药性的衍生物
公开(公告)号：WO2008067491A2
公开(公告)日：2008-06-05
申请号：PCT/US2007085974
申请日：2007-11-29
申请人： UNIV INDIANA RES & TECH CORP , MOLITORIS BRUCE A , BEDWELL DAVID M , SANDOVAL RUBEN M
发明人： MOLITORIS BRUCE A , BEDWELL DAVID M , SANDOVAL RUBEN M
IPC分类号： A61K31/7034
CPC分类号： A61K31/7036 , A61K31/7034
摘要： Various aspects related to the preparation of congeners of the aminoglycosides gentamicin such as the congener C2 and using this compound or derivatives thereof and pharmaceutically active salts to treat diseases that involve genetic mutations which introduce a missense or premature stop codon into a gene. Still other aspects include treating human or animal patients with the gentamicin congener C2 and derivatives and pharmaceutical salt thereof to overcome, or to at least mitigate, the symptoms of disease and disorders such as some forms of Becker's or Duchenne muscular dystrophy, Hurler's Syndrome and Cystic Fibrosis that have as their etiology the presence of a premature stop codon in a gene whose proper expression is necessary for good health.
摘要翻译：涉及制备氨基糖苷类庆大霉素如同系物C2的同系物的各个方面，并使用该化合物或其衍生物和药学活性盐来治疗引入遗传突变的疾病，其引入错义或过早终止密码子到基因中。还有其它方面包括用庆大霉素同类物C2及其衍生物和药物盐治疗人或动物患者以克服或至少减轻疾病和病症的症状，例如某些形式的贝克氏或杜氏肌营养不良症，霍乱综合征和囊性纤维化在其病因学中存在早期终止密码子的基因，其正常表达对于良好的健康是必需的。

5. 发明申请

WO1997034611A1 NOVEL CYSTIC FIBROSIS TREATMENT 审中-公开
标题翻译：新型CYSTIC FIBROSIS治疗
公开(公告)号：WO1997034611A1
公开(公告)日：1997-09-25
申请号：PCT/US1997003464
申请日：1997-03-21
申请人： UAB RESEARCH FOUNDATION , BEDWELL, David, M.
发明人： UAB RESEARCH FOUNDATION
IPC分类号： A61K31/70
CPC分类号： A61K31/7036 , A61K31/7048 , Y10S514/866
摘要： The present invention provides a method of stimulating expression of a functional full-length cystic fibrosis transmembrane conductance regulator protein in an individual in need of such treatment, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside. Also provided is a method of treating cystic fibrosis in an individual in need of such treatment, comprising the step of administering to said individual a therapeutically effective dose of an aminoglycoside. Further provided is a method of screening for a drug useful in the treatment of an individual having cystic fibrosis, comprising the step of determining said drug's ability to suppress premature stop mutations in a model of cystic fibrosis and a method of pharmacologically suppressing premature stop mutations in an individual having such mutations, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside.
摘要翻译：本发明提供了在需要这种治疗的个体中刺激功能性全长囊性纤维化跨膜传导调节蛋白的表达的方法，其包括向所述个体施用药理学有效剂量的氨基糖苷类的步骤。还提供了在需要这种治疗的个体中治疗囊性纤维化的方法，包括向所述个体施用治疗有效剂量的氨基糖苷的步骤。进一步提供筛选用于治疗患有囊性纤维化的个体的药物的方法，包括确定所述药物在囊性纤维化模型中抑制过早停止突变的能力的步骤，以及药理学上抑制过早停止突变的方法具有这种突变的个体，包括向所述个体施用药理学有效剂量的氨基糖苷的步骤。

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