COMPOSITIONS AND METHODS FOR THE TREATMENT OF JUVENILE NEURONAL CEROID LIPOFUSCINOSIS AND RELATED DISORDERS
    87.
    发明申请
    COMPOSITIONS AND METHODS FOR THE TREATMENT OF JUVENILE NEURONAL CEROID LIPOFUSCINOSIS AND RELATED DISORDERS 审中-公开
    治疗单纯性神经性白蛋白LIPOFUSCISOSIS和相关疾病的组合物和方法

    公开(公告)号:US20170049761A1

    公开(公告)日:2017-02-23

    申请号:US15251855

    申请日:2016-08-30

    Inventor: Tammy Kielian

    Abstract: Provided are materials and methods for the prevention and treatment of Juvenile Neuronal Ceroid Lipofuscinosis comprising administration of an effective amount of at least one of a hemi-channel inhibitor or a phosphodiesterase-4 inhibitor. In some embodiments, the methods comprise administration of an effective amount of each of a hemi-channel inhibitor and a phosphodiesterase-4 inhibitor. Also provided are pharmaceutical compositions comprising a hemi-channel inhibitor or a phosphodiesterase-4 inhibitor, as well as kits comprising at least one effective dose of a hemi-channel inhibitor or a phosphodiesterase-4 inhibitor or a combination of both.

    Abstract translation: 提供了用于预防和治疗少年神经元细胞脂褐色素沉积的材料和方法,包括施用有效量的半通道抑制剂或磷酸二酯酶-4抑制剂中的至少一种。 在一些实施方案中,所述方法包括施用有效量的每种半通道抑制剂和磷酸二酯酶-4抑制剂。 还提供了包含半通道抑制剂或磷酸二酯酶-4抑制剂的药物组合物,以及包含至少一种有效剂量的半通道抑制剂或磷酸二酯酶-4抑制剂或两者的组合的试剂盒。

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