肌萎缩侧索硬化症药物研发：回顾与展望
ALS drug development: reflections from the past and a way forward.
作者信息
Aggarwal Swati, Cudkowicz Merit
机构信息
Department of Neurology, Massachusetts General Hospital, Neurology Clinical Trials Unit, Charlestown, Massachusetts 02129, USA.
出版信息
Neurotherapeutics. 2008 Oct;5(4):516-27. doi: 10.1016/j.nurt.2008.08.002.
Abstract
Tremendous advances in our understanding of pathogenesis of amyotrophic lateral sclerosis (ALS) have provided a rich pipeline of drugs for clinical trialists. At least 32 unique compounds have been tested. Nevertheless, riluzole is currently the only treatment that prolongs survival. We present a critical overview of past clinical trials, how therapies are selected for testing in people, challenges with ALS clinical trial design and conduct, and ways to best move forward.
摘要
我们对肌萎缩侧索硬化症（ALS）发病机制的理解取得了巨大进展，为临床试验人员提供了丰富的药物研发渠道。至少32种独特的化合物已进行了测试。然而，利鲁唑是目前唯一能延长生存期的治疗方法。我们对过去的临床试验进行了批判性概述，介绍了如何选择用于人体测试的疗法、ALS临床试验设计与实施面临的挑战，以及未来最佳的推进方向。
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Design of phase II ALS clinical trials.肌萎缩侧索硬化症（ALS）II期临床试验的设计
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Human angiogenin is a neuroprotective factor and amyotrophic lateral sclerosis associated angiogenin variants affect neurite extension/pathfinding and survival of motor neurons.人血管生成素是一种神经保护因子，与肌萎缩侧索硬化相关的血管生成素变体影响运动神经元的神经突延伸/路径寻找和存活。
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Minocycline and riluzole brain disposition: interactions with p-glycoprotein at the blood-brain barrier.米诺环素和利鲁唑的脑内分布：与血脑屏障处P-糖蛋白的相互作用
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