ASBMR-RBDA Symposium on Rare Bone Diseases: Patients, Clinicians, and Scientists Working Together to Improve Lives

The ASBMR and Rare Bone Disease Alliance invite you to attend its 2023 Symposium on Thursday, October 12, 2023, one day prior the 2023 ASBMR Annual Meeting, at the Vancouver Convention Centre in Vancouver, BC, Canada. Organized by Drs. Michael Collins, Kathryn Dahir, Maegen Wallace, Leanne Ward, and Eric T. Rush, the goal of this topical meeting is to bring together experts in the field of rare bone disease research to present and discuss the latest advances in basic, translational, and clinical science.

Questions? Contact the ASBMR Business office by email at asbmr@asbmr.org or by phone at +1 (202) 367-1161. 

Program

Thursday, October 12, 2023
Vancouver Convention Centre, West Meeting Room 306

12:00 pm

Opening Keynotes

Chairs: Michael Collins, MD; NIH (United States) and Eric T. Rush, MD; Children's Mercy Kansas City (United States)

• Twenty Year Historical Perspective of Rare Bone Disease– Michael J. Econs, MD; Indiana University School of Medicine (United States)
• Patient and Patient Advocate: Experiences with Rare Bone Disease - Neena Nizar, CEO, The Janseen's Foundation (United States)

12:40 pm

Session I: Basic Science Informing Care

Chairs: Michael Collins, MD; NIH (United States) and Kelly Wentworth, MD; UCSF (United States)

• Translating insights from PTHR1 mechanistic studies to therapies for rare bone and mineral disease - Tom Gardella, PhD; Harvard University (United States)
• La protein: Manager of osteoclast multinucleation and resorption with preclinical efficacy in osteopetrosis - Jarred Whitlock, PhD; NIH (United States)
• Cellular and molecular mechanism underlying Fibrous Dysplasia - Yingzi Yang, PhD, Harvard University (United States)

Break

2:00 pm

Session II: Translational Advances in Rare Bone Disease Therapeutics

Chairs: Leanne Ward, MD, FRCPC; University of Ottawa (Canada) and Alison Boyce, MD; NIH (United States)

• A Rare Disease Informing a Common Disease: XLH and Degenerative Osteoarthritis - Carolyn Macica, PhD; Connecticut Children's Research Institute, United States
• Bench to Bedside Translation of Rare Disease Therapeutics; An Academic Perspective - Demetrios Braddock, MD; Yale University (United States)
• Achondroplasia: another step forward in treatments - Laurence Legeai-Mallet, PhD: INSERM U1163 (France)

3:00 pm

Session III: Latest in Clinical Trials

Chairs: Maegen Wallace, MD, MBA, FAOA, FAAOS; Phoenix Children’s (United States) and Danielle Gomez-Reynolds, MD; University of South Florida (United States) 

• Fibrodysplasia Ossificans Progressiva - Richard Keen, MBBS, PhD; Royal National Orthopaedic Hospital (United Kingdom)
• Ilofotase alpha and HPP - Lothar Seefried, MD; University of Wuerzburg (Germany)
• Emerging Therapies for Chronic Hypoparathyroidism:  Latest Clinical Trial Findings - Dolores Shobak, MD; UCSF (United States)

Break

4:20 pm

Session IV: Cutting-edge Research from Young Investigators

Chairs: Kathryn Dahir, MD; Vanderbilt University Medical Center (United States) and Eric T. Rush, MD; Children's Mercy Kansas City (United States)

• 4-6 oral presentations selected from the ASBMR 2023 Annual Meeting abstract submissions

5:00 pm – 6:30 pm

Poster Session and Reception

Supported  from educational funding donations  by Ascendis Pharma A/S, BioMarin Pharmaceutical Inc., Calcilytix Therapeutics, Inc. (a BridgeBio company), Inozyme Pharma, Ipsen Biopharmaceuticals, Inc., Kyowa Kirin, Inc., Regeneron Pharmaceuticals, Inc., and Ultragenyx Pharmaceutical Inc. 

(as of September 11, 2023).