SMALL MOLECULE THERAPEUTIC COMPOUNDS TARGETING THIOESTERASE DEFICIENCY DISORDERS AND METHODS OF USING THE SAME
    1.
    发明申请
    SMALL MOLECULE THERAPEUTIC COMPOUNDS TARGETING THIOESTERASE DEFICIENCY DISORDERS AND METHODS OF USING THE SAME 有权
    小分子治疗化学物质治疗精神病药物缺陷性疾病及其使用方法

    公开(公告)号:US20140148513A1

    公开(公告)日:2014-05-29

    申请号:US14110393

    申请日:2012-04-09

    IPC分类号: A61K31/133 A61K45/06

    摘要: The invention provides methods of inhibiting the development or progression of a thioesterase deficiency disorder in a mammal by the administration of a compound that functionally mimics the enzymatic activity of all thioesterases in mammals. Such thioesterase deficiency disorders include cancers and adult- or infant-neuronal ceroid lipofuscinoses (NCLs). The invention also provides small molecule mimics of thioesterases useful in the methods of the invention and pharmaceutical compositions containing the therapeutically effective compounds and methods of using the same.

    摘要翻译: 本发明提供了通过施用功能上模拟哺乳动物中所有硫酯酶的酶活性的化合物来抑制哺乳动物中硫酯酶缺乏症的发展或进展的方法。 这样的硫酯酶缺乏症包括癌症和成年或婴儿神经元样细胞脂褐素(NCL)。 本发明还提供了可用于本发明方法的硫酯酶的小分子模拟物和含有治疗有效化合物的药物组合物及其使用方法。