10th Annual SBP Rare Disease Day Symposium

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Faculty Host(s)
Yu Yamaguchi, M.D., Ph.D.
Regina Kapono
Date/Time: February 28, 2019 (8:45 AM - 5:00 PM)
Time Zone:
Event Location(s):
Building 12, SBP Auditorium

10905 Road to the Cure
La Jolla, CA 92121

Map Link

Check-in and continental breakfast begin at 8:00 AM

Rare Bone Disorders and Tumors:
New Frontiers of Translational Research

We proudly invite you to the 2019 SBP Rare Disease Day Symposium, "Rare Bone Disorders and Tumors: New Frontiers of Translational Research," Thursday, February 28, 2019 in La Jolla, CA. The symposium, chaired by Yu Yamaguchi, M.D., Ph.D. – Professor, Human Genetics Program, will be held at the Sanford Children's Health Research Center at Sanford Burnham Prebys Medical Discovery Institute's campus in La Jolla, CA. 

The 10th annual SBP Rare Disease Symposium will focus on rare bone disorders and bone tumors. Diseases to be covered will include multiple hereditary exostoses (MHE/MO), fibrodysplasia ossificans progressive (FOP), Ollier disease, achondroplasia, Larsen syndrome, osteogenesis imperfecta, chondrosarcoma, and Ewing sarcoma. The leaders in this field will discuss the recent progress in genetic, biological, and translational aspects of these diseases as well as the current situation of clinical trials for MHE and FOP.



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The 2019 SBP Rare Disease Day Symposium is presented by Sanford Burnham Prebys Medical Discovery Institute and made possible by patient advocacy and industry sponsorships, as well as private donations. Your free registration to the one-day symposium includes access to all scientific sessions, lunch, and light refreshments during conference breaks.


Please note that scheduled speakers are subject to change

Kristiina Vuori, M.D., Ph.D.
Sanford Burnham Prebys Medical Discovery Institute
Welcome Remarks

Keynote Speaker

Henry Kronenberg, M.D.
Massachusetts General Hospital and Harvard Medical School
Skeletal Stem Cells: Growth Plate and Beyond


David Feldman, M.D.
The Paley Orthopedic and Spine Institute
Factors in Improvement of Quality of Life in MHE: An Orthopedic Perspective

Maurizio Pacifici, Ph.D.
Children’s Hospital of Philadelphia
Mechanisms of Signaling Protein-Heparan Sulfate Interactions: Implications for MHE and FOP Pathogenesis

Eileen Shore, Ph.D.
University of Pennsylvania School of Medicine
Extra-Skeletal Bone Formation in FOP: A Multi-Step Process

Yu Yamaguchi, M.D., Ph.D.
Sanford Burnham Prebys Medical Discovery Institute
Towards Pharmacological Intervention of Osteochondromagenesis in MHE: Preclinical Evidence

Fei Shih, M.D.
Clementia Pharmaceuticals
Palovarotene as Potential Treatment for Fibrodysplasia Ossificans Progressiva and Multiple Osteochondroma: An Update

Judith Bovée, M.D., Ph.D.
Leiden University
Central and Peripheral Chondrosarcoma: Lessons Learned from Studying Their Benign Precursors

Benjamin Alman, M.D.
Duke University
Dysregulated  Growth Plate Development and Metabolism in the Etiology and Progression of Enchondromatosis

Adam Shlien, Ph.D.
The Hospital for Sick Children, Toronto
Formation Mechanisms of Canonical Gene Fusions in Bone and Soft Tissue Tumors

David Ornitz, M.D., Ph.D.
Washington University School of Medicine
Regulation of Osteogenic and Chondrogenic Growth Programs by FGF Signaling Pathways

John Graham, M.D.
Cedars-Sinai Medical Center and Harbor-UCLA Medical Center
A Clinician’s View of Larsen Syndrome and Related Disorders

Matthew Warman, M.D.
Boston Children’s Hospital and Harvard University
Preclinical Studies of Combination Therapies in Osteogenesis Imperfecta


What meals are included with my registration? 
Your free registration includes continental breakfast, lunch, and light refreshments during session breaks.

What are my transportation/parking options for getting to and from the event?
Sanford Children's Health Research Center is located 15 miles from the San Diego International Airport. Please note that public transportation in San Diego is limited and the hotel does not have a dedicated airport shuttle.

A one-way traditional taxi fare from the San Diego International Airport is approximately $60-80 with tip. Ride-sharing services such as Lyft and Uber are widely used in the San Diego area, offering fares from $25-80, depending on class of service.

Who do I contact with questions?
Event Inquiries: Regina Kapono,
Scientific Inquiries: Yu Yamaguchi,


We look forward to your participation!

Last Year’s Event – Congenital Disorders of Glycosylation and Deglycosylation