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COMPOSITIONS AND METHODS FOR THE TREATMENT OF JUVENILE NEURONAL CEROID LIPOFUSCINOSIS AND RELATED DISORDERS
COMPOSITIONS AND METHODS FOR THE TREATMENT OF JUVENILE NEURONAL CEROID LIPOFUSCINOSIS AND RELATED DISORDERS
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机译:治疗青少年神经性类胡萝卜脂蛋白病和相关疾病的组合物和方法
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摘要
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.
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