Bonescanada.org aims to empower healthcare professionals and patients dealing with childhood-onset rare bone disorders through collaboration, a multidisciplinary team of experts, and overcoming challenges like limited resources, integrating research and care, and facilitating technology and regulatory processes. They have enrolled over 400 children in their research program on conditions like Duchenne muscular dystrophy and osteogenesis imperfecta, using centralized imaging to support international clinical trials. Lessons from research also inform their clinical program and advocacy efforts.

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12. Bonescanada.org
Empowering health care professionals and the patient community
in the management of childhood-onset rare bone disorders

13. Collaboration with Patient Advocacy Groups

15. The Multi-Disciplinary Team of Experts
in Clinical Care and in Research

17. The challenges (not an exhaustive list)
• Human resourcing
• Physicians
• Allied Health Professionals
• Laboratory/diagnostic imaging personnel
• Space!
• For clinic visits with allied health professionals
• For research associates working with patients to collect data
• Integration of clinical trials into routine clinical care
• Minimize the separation between research and care
• Smooth logistically for patients and for health care providers
• Maximize support to clinical trial investigators to facilitate the Health Authority regulatory
documentation and process
• Technology
• Easy access to robust systems to collect, collate and output data
• Centralized REB process
• One application, one consent form, one reporting schema for a single study with multiple centres participating
• Development of registries to acquire long-term data from an academic-clinical lens
• Database management and statistical analysis

18. • Patient access to local and specialized care
• Research and knowledge translation
• Training ground for future clinicians and researchers

19. Lward@cheo.on.ca

21. Enrolled over 400 children and
followed t

22. CHILDHOOD-ONSET RARE BONE DISORDERS
The RESEARCH PROGRAM
• Boot-strapped to the CHEO clinical program in childhood-onset rare bone diseases
• Investigator-initiated trials and pharma-sponsored trials in conditions such as:
• Duchenne and Becker’s MD, osteogenesis imperfecta, X-linked hypophosphatemia, achondroplasia, steroid-
induced osteoporosis
• Strong emphasis currently on centralized skeletal imaging to support international clinical trials in rare disorders
• Patients have been enrolled in studies through the Ottawa site from across Canada
• Knowledge has led to clinical practice guidelines and drug development
The CLINICAL PROGRAM
• The lessons learned from the research realm are directly implemented into the CHEO Bone Disease Clinic and
communicated through publications and international clinical practice guidelines to clinicians around the world
The ADVOCACY PROGRAM
• The Canadian Consortium for Children’s Bone Health – educating health care providers
• The Canadian Alliance for Rare Disorders of the Skeleton – educating, engaging and empowering patients and
families

24. Website