Center for Human Experimental Therapeutics, University of Rochester School of Medicine and Dentistry, Rochester, New York, USA.
Mov Disord. 2012 Jan;27(1):31-41. doi: 10.1002/mds.23953. Epub 2011 Oct 13.
Huntington's disease (HD) is an inherited, progressive neurodegenerative disorder characterized by chorea, cognitive impairment, and behavioral disturbances. Despite advances in diagnosis and improved understanding of HD, treatment remains difficult due to challenging symptoms and a paucity of approved therapeutic interventions. Nonpharmacologic and pharmacologic strategies have been evaluated; regarding the latter, over 80 agents of various classes have been investigated in clinical trials or examined in case reports. Symptomatic treatment, however, is generally confined to antidopaminergic agents for motor dysfunction and antidepressants for mood disorders, while treatment for cognitive dysfunction remains vacant. Several different mechanisms to modify symptoms and disease progression have been targeted in clinical trials. This article reviews some of the more common pharmacologic treatments used for HD, discusses data regarding suboptimal agents that have been tested, and surveys treatments under investigation.
亨廷顿病(HD)是一种遗传性、进行性神经退行性疾病,其特征为舞蹈病、认知障碍和行为障碍。尽管在诊断方面取得了进展,对 HD 的认识也有所提高,但由于症状具有挑战性且批准的治疗干预措施有限,治疗仍然很困难。已经评估了非药物和药物策略;关于后者,已经在临床试验中研究了 80 多种不同类别的药物,并且在病例报告中也进行了检查。然而,对症治疗通常仅限于抗多巴胺能药物治疗运动功能障碍和抗抑郁药治疗情绪障碍,而认知功能障碍的治疗仍然空缺。在临床试验中已经针对几种不同的机制来改善症状和疾病进展。本文综述了一些用于 HD 的更常见的药物治疗方法,讨论了已测试的效果不佳的药物的数据,并调查了正在研究的治疗方法。