The epidemiology and treatment of ALS: focus on the heterogeneity of the disease and critical appraisal of therapeutic trials.
The epidemiology and treatment of ALS: focus on the heterogeneity of the disease and critical appraisal of therapeutic trials.
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DOI：
10.3109/17482968.2010.502940
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发表时间：
2011-01
期刊：
影响因子：
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通讯作者：
Eurals Consortium
中科院分区：
文献类型：
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作者：
Beghi E;Chiò A;Couratier P;Esteban J;Hardiman O;Logroscino G;Millul A;Mitchell D;Preux PM;Pupillo E;Stevic Z;Swingler R;Traynor BJ;Van den Berg LH;Veldink JH;Zoccolella S;Eurals Consortium
肌萎缩侧索硬化症(ALS)的有效治疗方法仍然难以捉摸。只有利鲁唑，一种被认为影响谷氨酸新陈代谢的药物，可以提高存活率，尽管程度不大。对治疗试验负面结果的解释包括可能的异质性，包括疾病易感性和致病机制，以及临床试验的错误方法。对这些因素的进一步了解将导致患者分层的改进，以及未来临床试验的设计。
Effective treatments for amyotrophic lateral sclerosis (ALS) have remained elusive. Only riluzole, a drug thought to affect glutamate metabolism, improves survival albeit to modest extent. Explanations for the negative results of therapeutic trials include a likely heterogeneity, both in disease susceptibility and pathogenic mechanisms, and faulty methodology of clinical trials. Further understanding of these factors will lead to improvements in patient stratification, and in the design of future clinical trials.