SIDE-CHAIN VARIANTS OF REDOX-ACTIVE THERAPEUTICS FOR TREATMENT OF MITOCHONDRIAL DISEASES AND OTHER CONDITIONS AND MODULATION OF ENERGY BIOMARKERS
申请人:Edison Pharmaceuticals, Inc.
公开号:US20160244392A1
公开(公告)日:2016-08-25
Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, stroke (MELAS), or Kearns-Sayre Syndrome (KSS) are disclosed, as well as compounds useful in the methods of the invention. Methods and compounds useful in treating other disorders are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.
本发明揭示了用于治疗或抑制线粒体疾病的方法,例如弗里德莱希共济失调症(FRDA)、勒伯遗传性视神经病变(LHON)、线粒体肌病、脑病、乳酸中毒、中风(MELAS)或科恩斯-萨耶综合症(KSS),以及在该方法中有用的化合物。本发明还揭示了用于治疗其他疾病的方法和化合物。本发明还揭示了用于评估受试者代谢状态和治疗效果的能量生物标志物。本发明还揭示了调节、规范或增强能量生物标志物的方法,以及用于该方法的化合物。