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补充材料介绍:肌萎缩侧索硬化症的治疗现状

Introduction to supplement: the current status of treatment for ALS.

作者信息

Miller Robert G, Appel Stanley H

机构信息

a Forbes Norris ALS Treatment and Research Center, California Pacific Medical Center , San Francisco , CA , USA and.

b Edwards Distinguished Endowed Chair for ALS Research; Chair, Department of Neurology; Co-Director , Houston Methodist Neurological Institute , Houston , TX , USA.

出版信息

Amyotroph Lateral Scler Frontotemporal Degener. 2017 Oct;18(sup1):1-4. doi: 10.1080/21678421.2017.1361447.

Abstract

ALS is a lethal neurodegenerative disease wherein the diagnosis is often delayed. Our understanding of the pathobiology is slowly expanding, and the number of new genes is rapidly increasing. The development of potential treatments targeting specific mechanisms is beginning to offer hope. Evidence-based treatments and the development of quality measures have raised the standard of care. The current status of treatment for ALS includes one drug riluzole that slows progression modestly, and another drug edaravone that was recently approved by FDA to slow ALS progression. Multidisciplinary clinics and symptomatic treatments ease the burden of ALS and prolong life. An overview of these treatments is provided here.

摘要

肌萎缩侧索硬化症(ALS)是一种致命的神经退行性疾病,其诊断往往会延迟。我们对病理生物学的理解正在缓慢扩展,新基因的数量也在迅速增加。针对特定机制的潜在治疗方法的开发开始带来希望。循证治疗和质量指标的发展提高了护理标准。ALS的当前治疗状况包括一种能适度减缓疾病进展的药物利鲁唑,以及另一种最近被美国食品药品监督管理局(FDA)批准用于减缓ALS进展的药物依达拉奉。多学科诊所和对症治疗减轻了ALS的负担并延长了生命。本文将对这些治疗方法进行概述。

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