9th International RASopathies Symposium

PROJECT SUMMARY Aberrant pathogenic signaling via the RAS-RAF-MEK-ERK signal transduction pathway is responsible for a large group of human congenital syndromes collectively termed RASopathies. These conditions result in many similar, but variable, clinical features and pose substantial medical, financial, and social burdens on families. The incidence of some RASopathies (e.g., Costello, cardiofaciocutaneous, Legius syndromes) is rare, whereas Noonan syndrome and Neurofibromatosis type 1 are estimated to affect ~1:2000 births. The study of rare diseases necessitates inclusion of broad expertise and partner networks to reach critical mass for productive insights. To advance understanding and treatments for the RASopathies, we propose to convene the 9th International RASopathies Symposium in Orlando, Florida on July 25-27, 2025. The format will center scientific sessions around organ systems that are understudied or emerging in significance. Sessions are designed to facilitate cross-disciplinary discussion beginning with a patient perspective, and basic, translational, and clinical research insights. The symposium is distinguished from others by being highly multidisciplinary, science-focused and inclusive of advocates and families, and distinct from targeted oncology or RAS-associated drug discovery meetings. The organizing committee consists of two parent advocates, an academic and a clinical chair. A broad group of presenters, panelists, and moderators, including early-stage investigators, hailing from academic, government, medical, advocacy, and industry sectors, will convene to discuss RASopathy genetics and predisposition to cancer, GI, neuro, cardio, and lymphatic systems, and the expanding therapeutic pipeline for RASopathies treatments. Conference advertisements will specifically welcome research trainees, clinical fellows, and new investigators from a broad range of disciplines to increase the talent pool from which to build an international network of researchers, benefitting the broader scientific and RASopathies communities. Funds are requested to support travel/lodging for organizers and speakers, scholarships for trainees/early-stage investigators whose meritorious abstracts are selected for presentation, and implementation of the conference logistics.