Department of Neurology, University Hospital of Bordeaux, Bordeaux, France.
Ther Adv Neurol Disord. 2010 Jul;3(4):249-63. doi: 10.1177/1756285610375328.
Multiple system atrophy (MSA) is a rare neurodegenerative disorder without any effective treatment in slowing or stopping disease progression. It is characterized by poor levodopa responsive Parkinsonism, cerebellar ataxia, pyramidal signs and autonomic failure in any combination. Current therapeutic strategies are primarily based on dopamine replacement and improvement of autonomic failure. However, symptomatic management remains disappointing and no curative treatment is yet available. Recent experimental evidence has confirmed the key role of alpha-synuclein aggregation in the pathogenesis of MSA. Referring to this hypothesis, transgenic and toxic animal models have been developed to assess candidate drugs for MSA. The standardization of diagnosis criteria and assessment procedures will allow large multicentre clinical trials to be conducted. In this article we review the available symptomatic treatment, recent results of studies investigating potential neuroprotective drugs, and future approaches for the management in MSA.
多系统萎缩（MSA）是一种罕见的神经退行性疾病，目前尚无任何有效治疗方法能够减缓或阻止疾病进展。其特征是多巴胺能药物反应不良性帕金森综合征、小脑性共济失调、锥体束征和自主神经衰竭，可任意组合出现。目前的治疗策略主要基于多巴胺替代治疗和改善自主神经衰竭。然而，症状管理仍然令人失望，目前尚无有效的治疗方法。最近的实验证据证实了α-突触核蛋白聚集在 MSA 发病机制中的关键作用。基于这一假说，已经开发出转基因和毒性动物模型来评估 MSA 的候选药物。诊断标准和评估程序的标准化将允许进行大型多中心临床试验。本文综述了目前的对症治疗方法、研究潜在神经保护药物的最新结果，以及 MSA 管理的未来方法。
