Lysosomal Disease Network Pilot Core

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.