Biomarkers for niemann-pick C disease and related disorders
    1.
    发明申请
    Biomarkers for niemann-pick C disease and related disorders 有权
    生物标记用于尼曼选择C型疾病及相关疾病

    公开(公告)号:US20090286272A1

    公开(公告)日:2009-11-19

    申请号:US12385529

    申请日:2009-04-10

    IPC分类号: C12Q1/02

    摘要: Methods for screening or diagnosing subjects for disorders involving accumulation of one or more oxysterols such as cytotoxic oxysterol accumulation, Niemann-Pick C(NPC) disease, lysosomal storage diseases, cholesterol trafficking diseases, and neurodegenerative diseases. Also provided are methods for methods for screening or diagnosing subjects (including infants and neonatal subjects) for NPC disease, methods for monitoring the progression, remission, and clinical status of NPC disease, and methods for evaluating the efficacy of therapeutic treatment of NPC disease.

    摘要翻译: 用于筛选或诊断涉及一种或多种氧固醇积累的疾病的受试者的方法,例如细胞毒素氧固醇积累,尼曼 - 皮克C(NPC)疾病,溶酶体贮积病,胆固醇运输疾病和神经变性疾病。 还提供了用于筛选或诊断NPC疾病的受试者(包括婴儿和新生儿受试者)的方法的方法,用于监测NPC疾病的进展,缓解和临床状态的方法以及评估NPC疾病治疗性治疗功效的方法。

    Biomarkers for Niemann-pick C disease and related disorders
    2.
    发明授权
    Biomarkers for Niemann-pick C disease and related disorders 有权
    尼曼选择C病和相关疾病的生物标志物

    公开(公告)号:US08497122B2

    公开(公告)日:2013-07-30

    申请号:US12385529

    申请日:2009-04-10

    IPC分类号: C12N5/071

    摘要: Methods for screening or diagnosing subjects for disorders involving accumulation of one or more oxysterols such as cytotoxic oxysterol accumulation, Niemann-Pick C(NPC) disease, lysosomal storage diseases, cholesterol trafficking diseases, and neurodegenerative diseases. Also provided are methods for methods for screening or diagnosing subjects (including infants and neonatal subjects) for NPC disease, methods for monitoring the progression, remission, and clinical status of NPC disease, and methods for evaluating the efficacy of therapeutic treatment of NPC disease.

    摘要翻译: 用于筛选或诊断涉及一种或多种氧固醇积累的疾病的受试者的方法,例如细胞毒素氧固醇积累,尼曼 - 皮克C(NPC)疾病,溶酶体贮积病,胆固醇运输疾病和神经变性疾病。 还提供了用于筛选或诊断NPC疾病的受试者(包括婴儿和新生儿受试者)的方法的方法,用于监测NPC疾病的进展,缓解和临床状态的方法以及评估NPC疾病治疗性治疗功效的方法。

    Novel Therapy for Treatment of Chronic Degenerative Brain Diseases and Nervous System Injury
    3.
    发明申请
    Novel Therapy for Treatment of Chronic Degenerative Brain Diseases and Nervous System Injury 有权
    治疗慢性退行性脑疾病和神经系统损伤的新型治疗方法

    公开(公告)号:US20080269183A1

    公开(公告)日:2008-10-30

    申请号:US11576125

    申请日:2005-09-27

    IPC分类号: A61K31/56 A61P25/00 A61P25/28

    CPC分类号: A61K31/57

    摘要: In one aspect the present invention provides neuroactive steroids for use in the treatment of nervous system disorders, degenerative brain diseases and congenital storage diseases. In a second aspect the invention provides neuroactive steroids in combination with a Liver X Receptor (LXR) ligand to effect treatment of a nervous system condition.

    摘要翻译: 一方面,本发明提供用于治疗神经系统疾病,退行性脑病和先天性储存疾病的神经活性类固醇。 在第二方面,本发明提供与肝X受体(LXR)配体组合的神经活性类固醇以实现神经系统状况的治疗。