4-(p-quinonyl)-2-hydroxybutanamide derivatives for treatment of mitochondrial diseases
    4.
    发明授权
    4-(p-quinonyl)-2-hydroxybutanamide derivatives for treatment of mitochondrial diseases 有权
    4-(对醌基)-2-羟基丁酰胺衍生物用于治疗线粒体疾病

    公开(公告)号:US07968746B2

    公开(公告)日:2011-06-28

    申请号:US12264838

    申请日:2008-11-04

    IPC分类号: C07C233/00

    摘要: Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, and stroke (MELAS), Kearns-Sayre Syndrome (KSS), are disclosed, as well as compounds useful in the methods of the invention, such as 4-(p-quinolyl)-2-hydroxybutanamide derivatives. Methods and compounds useful in treating other disorders such as amyotrophic lateral sclerosis (ALS), Huntington's disease, Parkinson's disease, and pervasive developmental disorders such as autism 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.

    摘要翻译: 还公开了治疗或抑制线粒体疾病如弗里德里希共济失调(FRDA),Leber氏遗传性视神经病变(LHON),线粒体肌病,脑病,乳酸脱落和中风(MELAS),Kearns-Sayre综合征(KSS)的方法 作为可用于本发明方法的化合物,例如4-(对 - 喹啉基)-2-羟基丁酰胺衍生物。 还公开了可用于治疗其他疾病如肌萎缩性侧索硬化(ALS),亨廷顿氏病,帕金森病和普遍发育障碍如自闭症的方法和化合物。 还公开了可用于评估受试者的代谢状态和治疗功效的能量生物标志物。 还公开了调节,归一化或增强能量生物标志物的方法以及可用于这些方法的化合物。