亨廷顿病标志物:改善临床效果
Huntington’s disease (HD) is a fatal, inherited, neurodegenerative disorder, characterized by chorea, motor instabilities, psychiatric manifestations and cognitive decline. Early genetic testing provides an opportunity for clinical interventions aimed at delaying onset and/or slowing progression of disease; however, current treatments for HD are limited, with only two FDA-approved drugs available to manage chorea. Encouragingly, however, several disease-modifying treatment approaches are in the therapeutic pipeline, with more than 200 clinical studies, and many more preclinical studies, in the works. Robust and reliable biomarkers are needed to predict disease onset, monitor disease progression and assess treatment responses. More specifically, biomarkers to stratify patients for clinical trials and biomarkers to track drug efficacy will certainly lead to improved clinical trial design and success. This book represents the first book focused solely on biomarkers for HD and represents a distinct resource that will be informative, not only for clinicians and those involved in clinical trial design, but also for a wide range of neurodegenerative disease researchers. This edited volume is written by top leaders in the field, and takes a cross-disciplinary approach to cover a broad spectrum of biomarker types, in order to provide the latest advances in the development of biochemical, molecular, imaging and digital biomarkers that have been investigated for HD. With the ultimate goal of treating patients, the development of disease-associated biomarkers has never been more important.
Huntington's病是一种致命的遗传性神经退行性疾病,特征为舞蹈症、运动不稳、精神心理症状以及认知衰退。早期的基因检测提供了一个机会来实现临床干预,旨在延缓疾病的发生和/或减缓疾病的进展;然而目前针对Huntington's病的治疗方法有限,仅有两种FDA批准的药物可用于管理舞蹈症。不过令人鼓舞的是,许多治疗手段正处在研发管道中,有超过200项临床研究正在进行中,而更多更广泛的前驱性研究也在进行当中。为了预测疾病的发作、监测疾病的发展以及评估治疗反应,需要有效的且可靠的生物标志物。具体来说,将患者进行分层以适应临床试验的生物标志物以及追踪药物疗效的生物标志物将会促进临床试验的设计与成功。这本书代表了对Huntington's病生物标志物的关注的第一本书,并为广泛的读者提供了有价值的资源——不仅适用于临床医生和负责设计临床试验的人,也适用于神经退行性疾病的广泛研究者。这一编辑版由该领域的顶级专家撰写,采取跨学科的方法来覆盖各种类型的生物标志物,以提供在Huntington's病中探究的最先进进展,包括生化、分子、影像以及数字生物标志物的发展最新成果。最终目标在于治疗患者,开发疾病相关的生物标志物对于这一过程来说从来没有像现在这样重要。
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