Compositions including small molecule mitofusin activators are described. The mitofusin activators are useful for treating diseases or disorders associated with a mitochondria-associated disease, disorder, or condition such as diseases or disorders associated with mitofusin 1 (MFN1) and/or mitofusin 2 (MFN2), or mitochondrial dysfunction. Methods of treatment, pharmaceutical formulations, and screening methods for identifying compounds that activate mitochondrial fusion are also described.
本文描述了包括小分子丝
裂霉素激活剂的组合物。丝裂
磷蛋白激活剂可用于治疗与线粒体相关的疾病或失调,如与丝裂
磷蛋白1(MFN1)和/或丝裂
磷蛋白2(MFN2)或线粒体功能障碍相关的疾病或失调。此外,还介绍了治疗方法、药物制剂以及用于鉴定激活线粒体融合的化合物的筛选方法。