Department of Molecular Neuroscience, UCL Institute of Neurology, London, UK.
J Neurochem. 2013 Aug;126 Suppl 1:125-41. doi: 10.1111/jnc.12322.
Friedreich's ataxia is a debilitating progressive neurodegenerative disease associated with cardiomyopathy and other features. The underlying cause is a deficiency of the mitochondrial protein frataxin which causes mitochondrial iron deposition, increased oxidative stress and impaired adenosine triphosphate production. Over the last 15 years, multiple clinical trials have assessed the efficacy of antioxidant agents in this disease. This article reviews trials of the two most important agents, namely co-enzyme Q10 and idebenone.
弗里德赖希共济失调是一种使人虚弱的进行性神经退行性疾病，与心肌病和其他特征有关。其根本原因是线粒体蛋白 frataxin 的缺乏，导致线粒体铁沉积、氧化应激增加和三磷酸腺苷生成受损。在过去的 15 年中，多项临床试验评估了抗氧化剂在这种疾病中的疗效。本文综述了两种最重要的药物，即辅酶 Q10 和依地酸二钠钙的临床试验。
