Lysosomal Disease Network Pilot Core

溶酶体疾病网络的试点/可行性核心将最大限度地发挥尖端创新研究的作用 为溶酶体疾病的研究人员提供机会，试图增进知识和提供 回答重要的、紧急的问题的方法是这个领域。核心的使命是支持 初步研究，是识别和测试新方法并生成 旨在测试即将实现的创新并促进早期发现的基础数据 有可能产生巨大影响的国家。因为这种赠款资助机制提供了内置的 在基础设施和行政支持方面，这些研究可以在最短的时间内迅速获得资金。通过 根据定义，试点研究旨在找出问题、不足之处和初步数据， 设计和/或实施完整的研究；它提供编写和获得完整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。试点研究应该推动 研究的包围圈，因此，核心是-也许矛盾的-兴奋的研究可能不会达到 意想不到的结果或不符合原始假设的目标。通过其目标：提供资金 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 在此应用程序中描述，并作为一种方式，使创新研究走在新的前沿 溶酶体疾病的治疗方法/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，这些项目包括：临床试验准备情况， 新生儿筛查、长期结果和全球覆盖面。

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.