President
CurrentThe most important thing I do in this position is support patients going through likely the toughest time of their lives with a complicated rare disease. What can I do to lighten the burden and help them understand what's coming on their health journey? It's different for everyone; though we share many of the same symptoms and long-term effects, our needs are unique even when going through the "same" disease. I run nearly all of the daily operations of the organization - communications, event planning, budgeting, fundraising, and all standard administrative tasks. I coordinate presentations and exhibition at multiple professional conferences annually. I support, update, and gather our Medical Advisory Board, Corporate Council, and Patient Advisory Committee for collaboration on research and events.One of the more outwardly impactful things we do is meet with our Patient Advisory Committee to identify areas of interest to patients: unmet needs, facts about what happens with our health long-term, etc, that are not currently represented in research. We then turn these conversations into questionnaires, which are well-received by our membership and often see a 10%+ response rate in a short period of time. With our data we then create posters, White Papers, and other educational documents to share with our community and attendees at these international conferences. The information produced from these "self-inquiries" is exciting new data for providers, researchers, and industry; there are several published papers that have been the result of tapping into our community's willingness to partner with the goal of advancing understanding of this disease. November 1, 2023 we were notified that we'd received a funding award from Critical Path Institute to work with them and NORD on a new US Cushing's patient registry that will collaborate with the ongoing RDCA-DAP (rare diseases drug accelerator platform). We will break ground on our registry build Jan 1, 2024.
