E-Publication E-Publication

Addressing Bone Manifestations of GD: The Latest Evidence in Evaluation and Treatment


Maximum Credits:


E-Publication E-Publication


Maximum Credits:
E-Publication E-Publication


Maximum Credits:

Overview: This accredited CME publication explores bone manifestations of Gaucher disease (GD) and includes video clips of expert faculty discussing key clinical considerations and patient case examples. Clinical evidence for GD treatment is also presented.

CME Information:

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
Miami, FL
Director, University Research Foundation for Lysosomal Storage Diseases, Inc. 
Boca Raton, FL
Medical Advisory Board, National Gaucher Foundation
Rockville, MD

Activity Planners
Julie Blum, PhD
Senior Clinical Content Manager
Baltimore, MD
Rebecca L. Julian, MS, ELS
Senior Manager, Editorial
Baltimore, MD
Samantha Gordon
CME Specialist
Baltimore, MD
Kathryn Schaefer, MSN, RN, CPHRM
Senior Manager, Accreditation and Compliance
East Lansing, MI

Lori Pender, PharmD, MPH, BCOP, ELS
Seattle, WA

Learning Objectives
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

Target Audience
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.
Providership Statement
Provided by Med-IQ.

Accreditation/Designation Statements
Med-IQ is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Med-IQ designates this enduring material for a maximum of 1.0 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Nurses, nurse practitioners, physician assistants, and other healthcare professionals who successfully complete the activity will receive a Statement of Participation indicating the maximum credits available.

Medium/Method of Participation
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
Disclosure Policy
Med-IQ requires any person in a position to control the content of an educational activity to disclose all relevant financial relationships with any commercial interest. The ACCME defines “relevant financial relationships” as those in any amount occurring within the past 12 months, including those of a spouse/life partner, that could create a conflict of interest (COI). Individuals who refuse to disclose will not be permitted to contribute to this CME activity in any way. Med-IQ has policies in place that will identify and resolve COIs prior to this educational activity. Med-IQ also requires faculty to disclose discussions of investigational products or unlabeled/unapproved uses of drugs or devices regulated by the US Food and Drug Administration.

Disclosure Statement
The content of this activity has been peer reviewed and has been approved for compliance. The faculty and contributors have indicated the following financial relationships, which have been resolved through an established COI resolution process, and have stated that these reported relationships will not have any impact on their ability to give an unbiased presentation. 
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.

Statement of Evidence-Based Content

Educational activities that assist physicians in carrying out their professional responsibilities more effectively and efficiently are consistent with the ACCME definition of continuing medical education (CME). As an ACCME-accredited provider of CME, it is the policy of Med-IQ to review and ensure that all the content and any recommendations, treatments, and manners of practicing medicine in CME activities are scientifically based, valid, and relevant to the practice of medicine. Med-IQ is responsible for validating the content of the CME activities it provides. Specifically, (1) all recommendations addressing the medical care of patients must be based on evidence that is scientifically sound and recognized as such within the profession; (2) all scientific research referred to, reported, or used in CME in support or justification of a patient care recommendation must conform to generally accepted standards of experimental design, data collection, and analysis.
Med-IQ is not liable for any decision made or action taken in reliance upon the information provided through this activity.

Contact Information 
For questions or comments about this activity or CPE contact hours, please contact Med-IQ. Call (toll-free) 866 858 7434 or email
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Med-IQ fully complies with the legal requirements of the ADA and the rules and regulations thereof. If any participant in this educational activity is in need of accommodations, please contact Med-IQ at 443 543 5200.
The information provided through this activity is for continuing education purposes only and is not meant to substitute for the independent medical judgment of a physician relative to diagnostic and treatment options of a specific patient’s medical condition.

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Acknowledgment of Commercial Support
This activity is supported by an educational grant from Sanofi Genzyme.

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Unless otherwise indicated, photographed subjects who appear within the content of this activity or on artwork associated with this activity are models; they are not actual patients or doctors.

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