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亨廷顿病:当前和未来的治疗前景。

Huntington's disease: Current and future therapeutic prospects.

机构信息

University of Rochester, USA and Clintrex LLC.

George Huntington Institute, Munster, Germany.

出版信息

Mov Disord. 2018 Jul;33(7):1033-1041. doi: 10.1002/mds.27363. Epub 2018 May 8.

DOI:10.1002/mds.27363
PMID:29737569
Abstract

Huntington's disease is a progressive neurodegenerative disorder for which therapies are woefully inadequate and do not prevent inevitable progression. Currently approved therapies are primarily aimed at treating chorea, but do not address the more clinically meaningful motor, behavioral, and cognitive features of the disease. However, there are a number of promising new therapies that are currently being studied in the laboratory, and in the clinic. This article will review the wide variety of therapies currently being tested, the advances in clinical trials and end points, and the many potentially relevant new targets. © 2018 International Parkinson and Movement Disorder Society.

摘要

亨廷顿病是一种进行性神经退行性疾病,目前的治疗方法远远不够,无法阻止其不可避免的进展。目前批准的治疗方法主要针对舞蹈病,但不能解决疾病更具临床意义的运动、行为和认知特征。然而,目前有许多有前途的新疗法正在实验室和临床中进行研究。本文将综述目前正在测试的各种治疗方法、临床试验和终点的进展,以及许多潜在相关的新靶点。© 2018 国际帕金森病和运动障碍学会。

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