This conference was held October 23-24, 2008 in Bethesda, Maryland.

Introduction

Program

Abstracts 

The 1st Advances in Rare Bone Diseases scientific conference took place October 23-24, 2008, in Bethesda, MD in the Natcher Center at the National Institutes of Health.

This conference brought together broad disciplines of biomedical science, experts in the various rare bone diseases, representatives from the FDA, the biopharmaceutical industry, and professional lay associations in support of furthering an understanding of the pathogenesis, natural history, and innovative treatment approaches for afflicted humans. Invited experts and attendees represented diverse disciplines that included genetics, molecular biology, nanobiology, endocrinology, rheumatology, nephrology, gastroenterology, nutrition, exercise physiology, orthopedics, radiology, anthropology, immunology, cell biology, and biomechanical engineering, among others.

The conference co-chairs, and sixteen international bone investigators serving as the Program Committee developed the 2-day scientific program that included overarching plenary lectures, directed state-of-the-art presentations, invited lectures from the Food and Drug Administration, the pharmaceutical industry involved in orphan diseases represented by rare bone diseases, and Hot Topic discussions for seven specific disease entities.

The objectives of the conference were:

(1) To examine the latest advances in basic, translational, and clinical research relating to a series of genetic bone diseases

(2) To understand how recent advances in science may be applied to the areas of bone biology and clinical osteology

(3) To allow selected trainees and junior faculty to present their relevant work to an audience of experts in the field

(4) To have an interested lay audience interact with experts in medicine, science and the biopharmacology industry interested in these bone diseases

(5) To forge new areas of understanding and need for research and therapeutics

Funding for this conference was made possible (in part) by 1R13AR056542-01 from the National Institute of Arthritis and Musculoskeletal and Skin Diseases, with co-funding from the National Institute of Diabetes and Digestive and Kidney Diseases, Office of the Director, National Institutes of Health, and the Office of Rare Diseases, National Institutes of Health. The views expressed in written conference materials or publications and by speakers and moderators do not necessarily reflect the official policies of the Department of Health and Human Services; nor does mention by trade names, commercial practices, or organizations imply endorsement by the U.S. Government.

Additional support for this conference was obtained in the form of educational grants from the American Society for Bone and Mineral Research; BioMarin Pharmaceutical, Inc.; Enobia Pharma, Inc.; Genzyme Corporation; Hope and Grace Foundation; International FOP Association; Kyowa Hakko Kirin Company, Limited; Orthopaedic Research Society.

PROGRAM COMMITTEE

• Michael J. Econs, MD, (Co-chair), Indiana University Department of Medicine
• Craig B. Langman, MD, (Co-chair), Northwestern University/Children's Memorial Hospital
• Peter H. Byers, MD, University of Washington
• Thomas O. Carpenter, MD, Yale University School of Medicine
• Michael Collins, MD, Skeletal Clinical Studies Unit, NIDCR
• Francis H. Glorieux, OC, MD, PhD, Shriners Hospital for Children, and Departments of Surgery, Pediatrics and Human Genetics, McGill University, Montreal, Canada
• Suzanne Jan de Beur, MD, The Johns Hopkins Bayview Medical Center
• Jeffrey C. King, MD, MSU, College of Human Medicine
• Frederick S. Kaplan, MD, University of Pennsylvania
• L. Lyndon Key, MD, Medical University of South Carolina
• Janice S. Lee, DDS, MD, MS, University of California
• Brendan Lee, MD, PhD, Baylor College of Medicine
• Arabella Leet, MD, Johns Hopkins Medicine
• Joan Marini, MD, PhD, Bone and Extracellular Matrix Branch, NICHD
• Geert Mortier, MD, Shriners Hospital for Children, and Departments of Surgery, Pediatrics and Human Genetics, McGill University, Montreal, Canada
• Horacio B. Plotkin, MD, FAAP, University of Nebraska School of Medicine
• Professor Peter S.N. Rowe, PhD, The Kidney Institute & Division of Nephrology, University of Kansas Medical Center
• Shlomo Wientroub, MD, Dana Children's Hospital, Tel Aviv Sourasky Medical Center, Israel
• Michael P. Whyte, MD, Shriners Hospital for Children

RBDPN NETWORK CO-CHAIRS

Kathleen D. Harper, Melorheostosis Association

Hillary Weldon, International Fibrodysplasia Ossificans Progressiva Association

USBJD PRESIDENT

J. Edward Puzas, PhD, University of Rochester Medical Center

ADVISOR

Charlene Waldman, The Paget Foundation