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    METHODS FOR IDENTIFYING SMALL MOLECULES THAT BIND SPECIFIC RNA STRUCTURAL MOTIFS
    1.
    发明公开
    METHODS FOR IDENTIFYING SMALL MOLECULES THAT BIND SPECIFIC RNA STRUCTURAL MOTIFS 审中-公开
    一种用于识别小的特定RNA的结构图案结合分子

    公开(公告)号：EP1377684A4

    公开(公告)日：2009-03-18

    申请号：EP02725663

    申请日：2002-04-11

    申请人： PTC THERAPEUTICS INC

    发明人： RANDO ROBERT ,  WELCH ELLEN

    IPC分类号： G01N27/62 ,  C12N15/09 ,  C12Q1/68 ,  G01N27/447 ,  G01N33/15 ,  G01N33/50 ,  G01N33/53 ,  G01N33/566 ,  C07H21/02 ,  G01N27/26

    CPC分类号： B82Y30/00 ,  G01N27/44726

    摘要： The present invention relates to a method for screening and identifying test compounds that bind to a preselected target ribonucleic acid ('RNA'). Direct, non-competitive binding assays are advantageously used to screen libraries of compounds for those that selectively bind to a preselected target RNA. Binding of target RNA molecules to a particular test compound is detected using any physical method that measures the altered physical property of the target RNA bound to a test compound. The structure of the test compound attached to the labeled RNA is also determined. The methods used will depend, in part, on the nature of the library screened. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads.

    PHARMACEUTICAL COMPOSITIONS AND SALTS OF A 1,2,4-OXADIAZOLE BENZOIC ACID
    2.
    发明公开
    PHARMACEUTICAL COMPOSITIONS AND SALTS OF A 1,2,4-OXADIAZOLE BENZOIC ACID 审中-公开
    PHARMAZEUTISCHE ZUSAMMENSETZUNGEN ANDD SALZE EINER 1,2,4-OXADIAZOLBENZOESÄURE

    公开(公告)号：EP3113619A4

    公开(公告)日：2017-09-13

    申请号：EP15757755

    申请日：2015-03-05

    申请人： PTC THERAPEUTICS INC

    发明人： DALI MANDAR V ,  TAKASUGI JAMES ,  WEETALL MARLA L ,  WELCH ELLEN

    IPC分类号： C07D271/06 ,  A61K9/00 ,  A61K31/4245

    CPC分类号： C07D271/06 ,  A61K9/0048 ,  A61K31/4245

    摘要： Provided herein are pharmaceutical compositions, which comprise a 1,2,4-oxadiazole benzoic acid or a pharmaceutically acceptable salt thereof. Further provided herein are certain pharmaceutically acceptable salts of a 1,2,4-oxadiazole benzoic acid and methods for making the same. Further provided herein are methods of treating or preventing a disease associated with a nonsense mutation or a premature stop codon, comprising administering such pharmaceutical compositions or pharmaceutically acceptable salts to a patient having a disease associated with a nonsense mutation or a premature stop codon.

    摘要翻译： 本文提供了药物组合物，其包含1,2,4-恶二唑苯甲酸或其药学上可接受的盐。 本文进一步提供了1,2,4-恶二唑苯甲酸的某些药学上可接受的盐及其制备方法。 本文进一步提供了治疗或预防与无义突变或提前终止密码子有关的疾病的方法，包括将所述药物组合物或可药用盐给予患有与无义突变或提前终止密码子相关的疾病的患者。