Lysosomal Disease Network Pilot Core

溶酶体疾病网络试点核心

• 批准号: 10264854
• 负责人: Chester B. Whitley
• 金额: $ 7.27万
• 依托单位: UNIVERSITY OF MINNESOTA
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-07-01 至 2025-07-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10264854
• 关键词: Area; Biological Markers; Clinical; Clinical Trials; Data; Disease; Disease Progression; Fostering; Foundations; Funding; Funding Mechanisms; Goals; Grant; Infrastructure; Knowledge; Lysosomal Storage Diseases; Methods; Mission; Monitor; Natural History; Neonatal Screening; Outcome; Pilot Projects; Research; Research Personnel; Research Project Grants; Technology; Testing; Time; Treatment outcome; Writing; clinical trial readiness; design; innovation; instrument; novel therapeutics; phase I trial

The Pilot/Feasibility Core for the Lysosomal Disease Network will maximize cutting-edge, innovative research opportunities for investigators in lysosomal diseases as an attempt to advance knowledge and provide approaches that answer important, emergent, questions is the field. The Core’s mission to support the preliminary research, is an extremely effective method to identify and test new methods and generate groundwork data designed to test innovations that are on the cusp of actualization, and to foster early findings that have the potential to have high impact. Because a grant funding mechanism of this sort provides built-in infrastructure and administrative support, these studies can be funded quickly with a minimum of lag time. By definition, a pilot study is intended to identify problems, areas of deficiency, and preliminary data, prior to design and/or implementation of a full study; it provides the preliminary data needed to write and obtain a full research project, and remains one of the most important stages of research. Pilot studies should push the research envelope and as such, the Core is—perhaps paradoxically—excited by studies may not attain outcomes that are unexpected or do not meet the goals of the original hypothesis. Through its Aims to: fund foundational, short-term research projects that are designed to test new methods, develop biomarkers, expand knowledge, and provide proof of concept findings, the Pilot/Feasibility Core will enhance the projects described in this application and serve as a way to enable innovative research at the forefront of new therapies/technologies/advancements for lysosomal disease. Pilot studies must also push forward the overarching themes of the LDN to fund research projects that include advances in: clinical trial readiness, newborn screening, long-term outcomes, and global reach.

溶酶体疾病网络的试点/可行性核心将最大限度地提高尖端的创新研究 为溶酶体疾病研究者提供机会，试图提高知识水平， 回答重要的、紧急的问题的方法就是这个领域。内核的使命是支持 初步研究，是一个非常有效的方法来识别和测试新的方法，并产生 基础数据，旨在测试即将实现的创新，并促进早期发现 有可能产生巨大影响的项目。因为这种赠款资助机制提供了内置的 如果有基础设施和行政支助，这些研究可以迅速获得资金，滞后时间最短。通过 定义，试点研究旨在确定问题，缺陷领域和初步数据， 设计和/或实施一个完整的研究;它提供了编写和获得完整的研究所需的初步数据。 研究项目，并仍然是最重要的研究阶段之一。试点研究应推动 因此，内核--也许是自相矛盾的--对可能无法达到的研究感到兴奋， 意外的或不符合原始假设目标的结果。其宗旨是： 基础性的短期研究项目，旨在测试新方法，开发生物标志物，扩大 知识，并提供概念验证结果，试点/可行性核心将加强项目 在本申请中所描述的，并作为一种方式，使创新的研究在新的前沿 溶酶体疾病的治疗/技术/进展。试点研究还必须推动 LDN的首要主题是资助研究项目，包括以下方面的进展：临床试验准备， 新生儿筛查、长期结果和全球覆盖范围。