PDF(Pubmed)
Abstract：
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by the loss of upper and lower motor neurons. Presently, three FDA-approved drugs are available to help slow functional decline for patients with ALS, but no cure yet exists. With an average life expectancy of only two to five years after diagnosis, there is a clear need for biomarkers to improve the care of patients with ALS and to expedite ALS treatment development. Here, we provide a review of the efforts made towards identifying diagnostic, prognostic, susceptibility/risk, and response fluid biomarkers with the intent to facilitate a more rapid and accurate ALS diagnosis, to better predict prognosis, to improve clinical trial design, and to inform interpretation of clinical trial results. Over the course of 20 + years, several promising fluid biomarker candidates for ALS have emerged. These will be discussed, as will the exciting new strategies being explored for ALS biomarker discovery and development.
摘要：
肌萎缩侧索硬化症（ALS）是一种致命的神经退行性疾病，其特征是上下运动神经元的丢失。目前,三种FDA批准的药物可以帮助减缓ALS患者的功能下降，但还没有治愈方法.诊断后平均预期寿命只有两到五年，显然需要生物标志物来改善ALS患者的护理并加快ALS治疗的发展.这里,我们回顾了为确定诊断所做的努力，预后，易感性/风险，和响应流体生物标志物，旨在促进更快速和准确的ALS诊断，为了更好地预测预后，为了改善临床试验设计，并告知临床试验结果的解释。在20多年的过程中，已经出现了几种有希望的ALS流体生物标志物候选物.这些将被讨论，正在探索的ALS生物标志物发现和开发的令人兴奋的新策略也是如此。
