Great Plains Rare Disease Summit - PROJECT SUMMARY In the United States, rare diseases are defined as diseases or conditions affecting less than 200,000 people, or approximately 1 in 1500 people. Although singularly rare, recent estimates suggest that there are over 10,000 rare disease that affect approximately 1 in every 10 Americans. People living with a rare disease often struggle to find or build patient support networks, or gain access to research opportunities given the rarity of some disorders. Sanford Research established the Coordination of Rare Diseases at Sanford (CoRDS) registry program to facilitate more meaningful interactions between patients, patent advocates, medical professionals and scientists, and has a rich 15 year history of hosting an annual rare disease symposium to bring together these key stakeholder groups. This event serves as a collaborative research summit that draws attendees from across the Great Plains region and beyond. With this proposal, our goal is to continue developing and growing the Great Plains Rare Disease Summit to provide access to the highest standard of scientific and clinical research in a format that is accessible to patients, advocates and community members. Hosting this summit in the Great Plains region will give patients and their families an opportunity to participate without the need for extensive or expensive travel, which can be a burden when living with high medical expenses and complex health issues. The mix of attendees is designed to facilitate exchanges between research and patient communities, with investigators able to witness first-hand how rare disease impacts patients, while patients, families, and advocates come away with a better scientific understanding of their conditions. Incorporating patient perspectives in the scientific process can lead to better clinical trial design and conduct which will ultimately lead to better patient outcomes. Our objective is to establish the Great Plains Rare Disease Summit as a destination conference for scientists, medical professionals, patients, advocates, and industry in the Great Plains region, and ultimately, throughout the US. The annual conference will promote groundbreaking research in rare diseases and provide an important avenue for conversation between scientists, clinicians, and persons living with the rare disease that they study and treat. We will achieve these objectives by 1) providing rural rare disease patients, caregivers and advocates access to a high quality rare disease education and support; 2) providing a forum for experts to present cutting edge research and build collaborative research efforts regionally and nationally; and 3) building up the rare disease community in the region for patients, caregivers, advocates, clinicians and scientists;
