Pilot and Feasibility Core

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PILOT & FEASIBILITY CORE- ABSTRACT/PROJECT SUMMARY The Frontiers in Congenital Disorders of Glycosylation Consortium's (FCGDC's) Pilot and Feasibility Core employs a dual pronged strategy for improving CDG patient care. On one hand, the core will recognize and fund cutting-edge CDG-focused research proposed by investigators that are new to the CDG field. The goal for this is to provide them with support (patients, samples, technical resources and funds) to generate preliminary data for a federal application. This will allow us to train and maintain a vibrant scientific and clinical workforce which is focused on improving CDG patient care. In parallel, we recognize that CDG is a rare disease and working in isolation (i.e. without patient or clinical context) may not yield clinically useful results. Hence, we will also develop a national level CDG-focused annual symposium that will bring together patients, CDG clinicians, CDG scientific experts and FCDGC pilot and feasibility awardees. The goal for this is to provide a singular platform where patient issues are discussed and morphed into sound research studies whose results could improve CDG patient care. We believe that implementing both of the above strategies will not only ensure a vibrant clinical and scientific workforce focused on CDG patient care but also accelerate the research findings into practical clinical care solutions for CDG patients.