Mechanistic and Therapeutic Advances in Rare Skeletal Disease

Osteogenesis Imperfecta Foundation, Inc., Gaithersburg MD

Investigators

Abstract

Project Summary Originally created as a patient advocacy network in 2006, the Rare Bone Disease Alliance (RBDA) has grown into a strong coalition focused on educating medical professionals, expanding research, and assisting patients and communities affected by rare skeletal diseases. RBDA scientific programs have cultivated an innovative and collaborative platform that tenaciously contributes to this body of research. The 2018 RBDA program, titled Mechanistic and Therapeutic Advances in Skeletal Diseases, will cover state of the art topics in diagnosis, preclinical mechanisms of disease, clinical targeting of signaling pathways, and novel clinical endpoints for assessment of efficacy. RBDA Scientific Meeting participants will include physician and scientific leaders, pharmaceutical industry representatives, patient advocates, and trainees working in the rare skeletal diseases field. The meeting will be a freestanding two day meeting that will precede the 2018 American Society for Bone and Mineral Research annual scientific meeting. The program proposes to: 1) Present and discuss cutting-edge approaches and largely unpublished data regarding (i) diagnostic techniques for rare skeletal diseases; (ii) preclinical models and pathogenesis for bone, cartilage, and craniofacial abnormalities; (iii) small molecule, protein, gene, and cell clinical therapies applicable to rare skeletal diseases; (iv) clinical target signaling pathways in the skeleton; (v) advances in endpoints and assessments in the context of clinical trials. 2) Pinpoint critical knowledge gaps and foster new research directions in the field of rare bone disease through robust dialogue and interactions among all meeting attendees. 3) Facilitate collaboration and network opportunities amongst all participants, including junior and senior investigators by thorough logistical planning and meeting promotion. 4) Maximize the involvement of underrepresented minorities, women, and individuals with disabilities who work in the rare bone disease arena by supporting their participation and travel to the meeting. With the support of new and continued partners that span multiple disciplines, the 2018 RBDA Scientific Meeting will encourage ambitious research agendas to advance the scientific understanding and clinical care for rare bone disease communities.