ASBMR-RBDA Three-Year Rare Bone Disease Symposia - The American Society for Bone and Mineral Research (ASBMR) is the largest professional, scientific and medical society established to bring together clinical and laboratory-based investigators who are involved in the study of bone, mineral and musculoskeletal disorders. Since 2002, the ASBMR has held one-day symposia preceding the society Annual Meeting that were funded by an R13 grant, tackled a specific topic and were very successful. Based on that strength, the ASBMR subsequently received 3-year R13 grants to systematically organize topical symposia for 2016-2018, 2019-2021 and 2022-2024. In that vein, the present application seeks a 3-year R13 grant to organize annual one-day symposia on Rare Bone Diseases, in partnership with the Rare Bone Disease Alliance. This is a critical and compelling field of research in urgent need of basic, transitional and clinical advances. The 2024, 2025 and 2026 symposia will respectively focus on: 1) “Exploring and Expanding Treatments and Analytical Tools in Rare Bone Diseases”; 2) “From the Genome to the Lived Experience”; 3) “A Review of the Rapidly Changing Landscape in Rare Disease Therapeutics”. The three symposia cover most pressing and timely areas of musculoskeletal research that are undergoing rapid advances in scientific knowledge and address disorders with major clinical morbidity, disability and mortality. The overall objective of this R13 is to share and discuss most recent findings and envision paths to translation and clinical testing of new therapies, with the goal of improving care of patients with rare bone diseases. The symposia will gather the best researchers, present state-of-the-art findings in each topic area, foster interactions and collaborations between young and established investigators, and chart future goals and directions. The symposia will also provide a forum to learn of novel strategies pursued in other research fields and their potential to expand rare bone disease research and therapeutic horizons. Symposia attendees will be encouraged to stay and take part in the Annual Meeting to further interact with musculoskeletal researchers, establish additional connections and learn from common bone diseases. Programs for all three symposia were developed by an organizing committee, and speakers for the first two symposia have confirmed their participation. Presenters will include established and young investigators, women and men, and national and international participants. There will be poster sessions and preference will be given to young investigators to present their abstract orally to further facilitate and encourage direct interactions between young and senior investigators. The 2024 symposium will discuss latest advances on the pathogenesis and potential treatments of rare bone diseases and will explore novel therapeutic strategies and future analytical tools by including experts from academia, industry and government entities. In 2025, the symposium will go from bench to bedside and feature an array of speakers who will be covering topics that span the genome to the patient voice through their lived experiences. The 2026 symposium will review the rapidly changing landscape of rare bone disease therapeutics and feature a diverse group of speakers including patients, researchers from academia and industry, and government officials.
