Clinical Core

项目总结/摘要 临床核心提供服务和专业知识，并有一个强大的培训组成部分囊性纤维化 (CF)research.这些服务侧重于监管支持、研究设计和开展支持。我们作为 为合作者提供先进的临床试验设计和实施的本地，国家和国际资源， 生物统计学和流行病学方法以及包括成本效益在内的比较效应研究方法 分析.核心支持UW囊性纤维化研究和翻译中心的中心目标 （CFRTC）：使用高效调节剂治疗（HEMT）启动，以发现囊性变的新方面， 纤维化（CF）的病理生理学，定义临床表现和最佳治疗方法的“新” CF将成为的疾病，并对持续存在的疾病方面进行基础和转化研究 尽管HEMT。此外，CC促进CFRTC生物医学核心之间的合作， 通过共享现有和发展的扩展的人类和细菌的转化研究 与临床数据库链接的储存库，能够开发改进的测定和临床结果 措施我们有能力通过药物开发继续推进新疗法 管道和过渡到CF的主要研究，重点是提高我们对疾病的理解， 先进的治疗方法的设置-特别是HEMT，将永远改变CF的临床景观。

PROJECT SUMMARY/ABSTRACT The Clinical Core provides both services and expertise and has a strong training component for cystic fibrosis (CF) research. The services focus on regulatory support, and study design and conduct support. We serve as a local, National and International resource to collaborators for advanced clinical trial design and conduct, biostatistical and epidemiologic methods and comparative effect research methods including cost-effectiveness analysis. The Core supports the central aims of the UW Cystic Fibrosis Research and Translation Center (CFRTC): to use highly effective modulator therapy (HEMT) initiation to discover novel aspects of cystic fibrosis (CF) pathophysiology, define the clinical manifestations and best treatment approaches for the “new” disease that CF will become, and perform basic and translational research on disease aspects that persist despite HEMT. Further, the CC fosters collaboration across the CFRTC biomedical cores to enhance translational research through the sharing of existing and development of expanded human and bacterial repositories linked to clinical databases that enable development of improved assays and clinical outcome measures. We are well positioned to continue to advance novel therapeutics through the drug development pipeline and transition to major studies in CF that focus on improving our understanding of the disease in the setting of advancing therapeutics – specifically HEMT that will forever change the clinical landscape in CF.