Landis Outstanding Mentor Award Supplement

项目摘要 肌萎缩侧索硬化症（ALS）是一种导致肌肉进展的致命神经退行性疾病 虚弱和吞咽障碍（吞咽困难）。吞咽困难会导致营养不良，脱水， 气管吸入肺炎和死亡父母补助金的主要动机是 阻碍ALS吞咽困难临床护理的知识缺口。总体目标是改善临床 通过1）增加我们对吞咽困难的控制机制和进展速度的理解 在ALS中，和2）识别吞咽困难的敏感临床标志物。我们将表演一系列的器乐 在100名ALS患者中进行吞咽评估，并提供关键的纵向数据，以帮助建立 第一个ALS吞咽下降的时间过程模型。需要这样一个模式来指导最佳做法 建议、干预的最佳时机、未来临床试验的规划和设计，以及 实验治疗效果的解释。我们还将测试的判别能力和临床效用， 一组有前途的吞咽下降的临床标志物， 传播到ALS临床环境中。我们的长期目标是改善吞咽的临床护理 ALS中的疾病这项研究将为研究脑梗死的病理生理机制提供新的见解。 ALS患者不安全和低效的吞咽，以推动未来干预策略的发展， 更早、更准确地识别吞咽障碍。更早识别，更好 ALS中吞咽功能障碍的治疗策略将导致口服摄入，营养， 肺健康和生活质量，并最终降低吸入性肺炎相关的死亡率， 挑战人口。这兰迪斯补充奖将支持培训和发展后- 博士研究员，他将致力于这项家长补助金。它还将支持受训人员的职业发展 活动

Project Summary Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease causing progressive muscle weakness and impairments in swallowing (dysphagia). Dysphagia leads to malnutrition, dehydration, tracheal aspiration, pneumonia, and death. The parent grant is strongly motivated by fundamental knowledge gaps that hinder clinical care of dysphagia in ALS. The overarching goal is to improve clinical practice by 1) increasing our understanding of governing mechanisms and progression rates of dysphagia in ALS, and 2) identification of sensitive clinical markers of dysphagia. We will perform serial instrumental swallowing evaluations in 100 individuals with ALS and provide critical longitudinal data to help establish the first time-course model of ALS swallowing decline. Such a model is needed to guide best practice recommendations, optimal timing of interventions, planning and design of future clinical trials, and interpretation of experimental treatment effects. We will also test the discriminant ability and clinical utility of a set of promising clinical markers of swallowing decline that are pragmatically designed for easy dissemination into ALS clinical settings. Our long-term goal is to improve clinical care of swallowing disorders in ALS. The proposed study will deliver new insights into the pathophysiologic mechanisms of unsafe and inefficient swallowing in ALS to drive the development of future intervention strategies and lead to earlier and more accurate identification of swallowing impairment. Earlier identification and better treatment strategies for swallowing dysfunction in ALS will lead to improvements in oral intake, nutrition, pulmonary health and quality of life, and ultimately reduce aspiration pneumonia associated mortality in this challenging population. This Landis Supplement Award will support the training and development of a post- doctoral fellow, who will work on this parent grant. It will additionally support trainee career development activities.