Mechanistic and Therapeutic Insights into Skeletal Biology Learned from the Study of Rare Bone Diseases

George R. Brown Convention Center, 1001 Avenida De Las Americas, Houston, TX 

The Mechanistic and Therapeutic Insights into Skeletal Biology Learned from the Study of Rare Bone Diseases Workshop Summary: To date, nearly 500 unique rare bone diseases have been described, which in the aggregate affect 1 in 5,000 live births, and comprise 5% of all birth defects. Typically the disability caused by these diseases affects individuals throughout their lives. Currently, the vast majority of these disorders have no cure, yet studying rare bone diseases has helped elucidate biological pathways and reveal valuable targets for drug discovery that are benefitting patients with common bone diseases too.

 

This workshop, coordinated by the National Bone Health Alliance (NBHA) in partnership with the Rare Bone Disease Patient Network and with the support of the American Society for Bone and Mineral Research (ASBMR) and the United States Bone and Joint Initiative (USBJI) aims to:

 

1) Convene a multi-disciplinary group of experts including researchers, clinicians, and patients with rare bone diseases to advance our understanding of skeletal biology and encourage the development of novel therapies to improve outcomes for individuals with both common and rare bone diseases;

 

2) Identify gaps in our knowledge regarding the biologic mechanisms underlying rare bone disorders and share effective research strategies in translational and therapeutic areas;

 

3) Encourage broad participation both by holding the event the day before the ASBMR 2014 Annual Meeting and providing funding for a significant number of young investigators to attend; and

 

4) Disseminate the results broadly by publishing the proceedings of the program in a peer-reviewed journal.

 

The one-day workshop will include lectures by invited speakers, and multiple opportunities to initiate collaborations and encourage future research partnerships.