Rare bone disease: Highlights from ASBMR 2022 - Part 3

In this third episode of a three-episode podcast series, COR2ED and ASBMR have partnered with patient advocates Inês Alves (European Rare Bone Forum) and Tracy Hart (Osteogenesis Imperfecta Foundation) to provide rare bone disease highlights from the ASBMR 2022 Annual Meeting, focusing on the patient perspective. The experts opened the podcast with the importance of engaging diverse patient communities. Independent of geography, patients should be educated about their condition and prepared for clinical trial participation; advocacy groups play a central role in this engagement. The experts then discussed outcomes of the IMPACT survey in OI (osteogenesis imperfecta), whose extensive database (66 countries, >2000 participants) offers meaningful insights into the clinical, humanistic and economic challenges faced by OI patients around the world. Looking to advances in pre-clinical research, the experts reviewed data on nanoparticle-mediated delivery of small interfering RNA (siRNA) to targeted mutation sites, tested in an animal model of autosomal dominant osteopetrosis. As the experts note, patient communities struggle with lengthy research and development timelines for novel therapies; therefore, it’s important for advocates to keep communities informed about ongoing efforts.