Addressing Bone Manifestations of GD: The Latest Evidence in Evaluation and Treatment
Pramod K. Mistry, MD, PhD, FRCP
Professor of Medicine and Pediatrics
Professor of Cellular & Molecular Physiology
Director, National Gaucher Disease Treatment Center
Yale University School of Medicine
Department of Internal Medicine
New Haven, CT
Neal J. Weinreb, MD, FACP
Voluntary Associate Professor of Clinical Genetics and Medicine (Hematology)
Miller School of Medicine, University of Miami
Director, University Research Foundation for Lysosomal Storage Diseases, Inc.
Boca Raton, FL
Medical Advisory Board, National Gaucher Foundation
Julie Blum, PhD
Senior Clinical Content Manager
Rebecca L. Julian, MS, ELS
Senior Manager, Editorial
Kathryn Schaefer, MSN, RN, CPHRM
Senior Manager, Accreditation and Compliance
East Lansing, MI
Lori Pender, PharmD, MPH, BCOP, ELS
Upon completion, participants should be able to:
- Recognize the relationship between bone physiology and GD
- List the diagnostic tools critical for the identification of bone involvement in GD
- Determine appropriate GD treatment strategies for patients with bone-related GD manifestations
This activity is intended for genetic counselors, geneticists, and hematologists.
Statement of Need
The majority of patients with type 1 GD experience bone manifestations, which can be associated with intense pain, impaired mobility, and reduced quality of life. Recent evidence has provided new insight in diagnosing and monitoring bone disease progression. In addition, clinical trials have shown that enzyme replacement therapy (ERT) and substrate reduction therapy (SRT) may be beneficial for mitigating bone-related complications of GD. As data regarding the genetics, biochemistry, and pathophysiology of GD-related bone manifestations continue to emerge, clinicians, researchers, and geneticists need to be aware of advances in the clinical evidence and the application of that knowledge to the care of patients with GD.
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This CME activity consists of a 1.0-credit online publication. To receive credit, read the introductory CME material, read the publication, and complete the post-survey, evaluation, attestation, and post-test, answering at least 70% of the post-test questions correctly.
Initial Release Date: October 10, 2018
Expiration Date: October 9, 2019
Estimated Time to Complete This Activity: 1 hour
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Pramod K. Mistry, MD, PhD, FRCP
Consulting fees/advisory boards: Sanofi Genzyme
Fees received for promotional/non-CME activities: Sanofi Genzyme
Contracted research: Sanofi Genzyme
Neal J. Weinreb, MD, FACP
Consulting fees/advisory boards: Aeglea BioTherapeutics, Pfizer, Inc., Sanofi Genzyme
The writer, peer reviewers, and activity planners have no financial relationships to disclose.
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This activity is supported by an educational grant from Sanofi Genzyme.
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