Division of Child Neurology, Childrens Hospital of Pittsburgh of UPMC, Pittsburgh, PA 20892, USA.
Neurotherapeutics. 2013 Apr;10(2):320-8. doi: 10.1007/s13311-012-0175-0.
The incidence of mitochondrial diseases has been estimated at 11.5/100,000 (1:8500) worldwide. In the USA up to 4000 newborns annually are expected to develop a mitochondrial disease. More than 50 million adults in the USA also suffer from diseases in which primary or secondary mitochondrial dysfunction is involved. Mitochondrial dysfunction has been identified in cancer, infertility, diabetes, heart diseases, blindness, deafness, kidney disease, liver disease, stroke, migraine, dwarfism, and resulting from numerous medication toxicities. Mitochondrial dysfunction is also involved in normal aging and age-related neurodegenerative diseases, such as Parkinson and Alzheimer diseases. Yet most treatments available are based on empiric data and clinician experience because of the lack of randomized controlled clinical trials to provide evidence-based treatments for these disorders. Here we explore the current state of research for the treatment of mitochondrial disorders.
线粒体疾病的发病率估计为全球每 100000 人中有 11.5 例（1:8500）。在美国，每年预计有多达 4000 名新生儿会患上线粒体疾病。美国也有超过 5000 万成年人患有原发性或继发性线粒体功能障碍相关疾病。线粒体功能障碍已在癌症、不孕、糖尿病、心脏病、失明、耳聋、肾病、肝病、中风、偏头痛、侏儒症以及许多药物毒性中得到确认。线粒体功能障碍也与正常衰老和与年龄相关的神经退行性疾病有关，如帕金森病和阿尔茨海默病。然而，由于缺乏随机对照临床试验为这些疾病提供基于证据的治疗方法，大多数可用的治疗方法都是基于经验数据和临床医生的经验。在这里，我们探讨了治疗线粒体疾病的研究现状。
