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    METHODS FOR TREATING EPILEPSY
    1.
    发明申请
    METHODS FOR TREATING EPILEPSY 审中-公开
    治疗癫痫的方法

    公开(公告)号：WO2017075312A1

    公开(公告)日：2017-05-04

    申请号：PCT/US2016/059262

    申请日：2016-10-28

    申请人： PTC THERAPEUTICS, INC.

    发明人： WELCH, Ellen ,  TOMIZAWA, Yuki ,  VAZIRANI, Priya

    IPC分类号： A61K31/5377 ,  A61K31/4245

    CPC分类号： A61K31/535

    摘要： Provided herein are methods of treating, preventing, ameliorating or managing a nonsense mutation mediated epileptic disease, comprising administering a 1,2,4-oxadiazole benzoic acid to a patient having a nonsense mutation mediated epileptic disease. In particular, provided herein are methods of treating, preventing, ameliorating or managing a CDKL5 and/or SCN1A (Dravet syndrome) nonsense mutation mediated epileptic disease.

    摘要翻译： 本文提供了治疗，预防，改善或控制无义突变介导的癫痫疾病的方法，其包括向患有无义突变介导的癫痫疾病的患者施用1,2,4-恶二唑苯甲酸。 具体而言，本文提供了治疗，预防，改善或控制CDKL5和/或SCN1A（Dravet综合征）无义突变介导的癫痫疾病的方法。

    SYNTHESIS AND IDENTIFICATION OF NOVEL RSK-SPECIFIC INHIBITORS
    6.
    发明申请
    SYNTHESIS AND IDENTIFICATION OF NOVEL RSK-SPECIFIC INHIBITORS 审中-公开
    新型RSK特异性抑制剂的合成与鉴定

    公开(公告)号：WO2011019995A3

    公开(公告)日：2012-06-21

    申请号：PCT/US2010045440

    申请日：2010-08-13

    申请人： HECHT SIDNEY M ,  LANNIGAN-MACARA DEBORAH A ,  SMITH JEFFREY ALLAN ,  O'DOHERTY GEORGE O ,  HILINSKI MICHAEL KENNETH

    发明人： HECHT SIDNEY M ,  LANNIGAN-MACARA DEBORAH A ,  SMITH JEFFREY ALLAN ,  O'DOHERTY GEORGE O ,  HILINSKI MICHAEL KENNETH

    IPC分类号： A61K31/535 ,  A61K31/075

    CPC分类号： A61K31/535 ,  A61K31/353 ,  A61K31/7048 ,  C07H17/075

    摘要： A composition comprising an SL0101 [kaempferol 3-O-(3",4"-di-O-acetyl-a-L- rhamnopyranoside)] derivative compound that has enhanced ability to inhibit RSK activity, relative to the parent compound is provided. The compounds have utility for treating any disease or conditions characterized or associated with excess or undesirable RSK activity. For example the RSK inhibitors of the present invention can be used to reduce the proliferation of neoplastic cells or for inhibiting the establishment or maintenance of an intracellular pathogenic infection by pathogens whose pathogenicity derives in part from the pathogen's ability to impede endosomal/phagosomal maturation in the host cell.

    摘要翻译： 提供了包含相对于母体化合物具有增强的抑制RSK活性的能力的SL0101 [山奈酚3-O-（3“，4” - 二-O-乙酰基-α-L-鼠李糖吡喃糖苷）]衍生物的组合物。 该化合物可用于治疗任何特征性或与过量或不期望的RSK活性相关的疾病或病症。 例如，本发明的RSK抑制剂可用于减少肿瘤细胞的增殖或用于抑制病原体的细胞内病原体感染的建立或维持，其致病性部分源于病原体阻碍内源性/吞噬体成熟的能力 宿主细胞。

    METHOD OF TREATING A VIRAL INFECTION DYSFUNCTION BY DISRUPTING AN ADENOSINE RECEPTOR PATHWAY
    8.
    发明申请
    METHOD OF TREATING A VIRAL INFECTION DYSFUNCTION BY DISRUPTING AN ADENOSINE RECEPTOR PATHWAY 审中-公开
    通过破坏腺苷受体途径治疗病毒感染功能失调的方法

    公开(公告)号：WO2012012736A3

    公开(公告)日：2012-03-15

    申请号：PCT/US2011045035

    申请日：2011-07-22

    申请人： UNIV OHIO STATE ,  DAVIS IAN C

    发明人： DAVIS IAN C

    IPC分类号： A61K31/00

    CPC分类号： A61K31/7076 ,  A61K31/00 ,  A61K31/138 ,  A61K31/275 ,  A61K31/277 ,  A61K31/416 ,  A61K31/52 ,  A61K31/522 ,  A61K31/535

    摘要： Described herein is a method of treating a viral infection such as an influenza infection, in a subject comprising administering an effective amount of a pharmaceutical composition to disrupt a adenosine receptor pathway, such as the Aradenosine receptor pathway, in a subject. The adenosine receptor pathway includes the steps of 1) producing the adenosine precursor adenosine triphosphate (ATP), 2) releasing ATP into the extracel lular space, 3) enzymatic conversion of ATP to adenosine, 4) activation of the adenosine receptor and the adenosine receptor cascade, and 5) clearance of adenosine from the extracellular space by degradation or uptake into a cell. The method includes affecting at least one of these steps so as to decrease the activation of the adenosine receptor pathway. This may be accomplished by decreasing the production, release, or conversion of ATP to adenosine, decreasing the expression of the adenosine receptor, antagonizing adenosine receptor activation, and/or increasing adenosine clearance.

    摘要翻译： 本文描述了治疗受试者中的病毒感染例如流感感染的方法，其包括施用有效量的药物组合物以破坏受试者中的腺苷受体途径，例如Aradenosine受体途径。 腺苷受体途径包括以下步骤：1）产生腺苷前体腺苷三磷酸（ATP），2）将ATP释放到胞外空间中，3）将ATP酶转化成腺苷，4）腺苷受体和腺苷受体的活化 级联，和5）通过降解或摄入细胞从细胞外空间清除腺苷。 该方法包括影响这些步骤中的至少一个以降低腺苷受体途径的活化。 这可以通过减少ATP产生，释放或ATP转化为腺苷，降低腺苷受体的表达，拮抗腺苷受体活化和/或增加腺苷清除来实现。