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Manifestations of Gaucher Disease: Rare or Under-recognized?

Premiere Date: Thursday, February 23, 2023

This activity offers CE credit for:

  1. ABIM (MOC)
  2. Medicine (accme)
  3. Nursing (ANCC)
  4. Pharmacy (acpe)
  5. PA (aapa)
  6. Other

All other clinicians will receive a Certificate of Attendance stating this activity was certified for AMA PRA Category 1 Credit™

Credit Expiration Date:
Friday, February 23, 2024
Note: Credit Is No Longer Available


Tamanna Roshan Lal, MB, ChB, FAAP, FACMGTamanna Roshan Lal, MB, ChB, FAAP, FACMG (Moderator)
Medical Geneticist
Director of Clinical Trials
Rare Disease Institute, Children's National Hospital
Washington, DC

Coy Heldermon, MD, PhDCoy Heldermon, MD, PhD 
Associate Professor
Director of the Pediatric and Adult Multi-Disciplinary Lysosomal Storage Disease Clinics
Gainesville, FL

Dimitri Krainc, MD, PhDDimitri Krainc, MD, PhD 
Chair, Department of Neurology
Director, Simpson Querrey Center for Neurogenetics
Aaron Montgomery Ward Professor
Northwestern University Feinberg School of Medicine
Chicago, IL

Neal J. Weinreb, MD. FACPNeal J. Weinreb, MD. FACP 
Voluntary Associate Professor
Department of Human Genetics
University of Miami Miller School of Medicine
Miami, FL

Statement of Need

Gaucher disease (GD) is a genetic disorder that causes fatty substances to accumulate to harmful levels in organs and tissues, often resulting in hepatosplenomegaly, anemia, thrombocytopenia, bone pain and fractures, and conditions like arthritis. GD is rare, and even specialists struggle to recognize and diagnose the condition. Delays to initiation of therapy are common and can result in irreversible complications.

During this 60-minute recorded symposium, expert panelists will discuss GD, including its less commonly reported manifestations, diagnostic pathways, and patient-centric multidisciplinary management of the disorder.

Learning Objectives

At the end of this CE activity, participants should be able to:

  • Identify under-recognized clinical manifestations of GD.
  • Evaluate the efficacy of current diagnostic methods in recognizing rare manifestations in GD.
  • Implement patient-centric management strategies within a multidisciplinary team to optimize long-term outcomes of patients with GD.

Financial Support

Supported by an educational grant from Takeda Pharmaceuticals U.S.A., Inc.

Target Audience

Physicians, PAs, nurse practitioners, PCPs, nurses, pharmacists, genetic counselors, and pediatricians, specializing in hematology, neurology, hepatology, gastroenterology, pulmonologists, radiology, and orthopedic surgery.

Credit Information

ABIM MOC Credit:
Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 medical knowledge MOC points in the American Board of Internal Medicine's (ABIM) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABIM MOC credit.

Learning Formats
Enduring Material

Royal College MOC:
Through an agreement between the Accreditation Council for Continuing Medical Education and the Royal College of Physicians and Surgeons of Canada, medical practitioners participating in the Royal College MOC Program may record completion of accredited activities registered under the ACCME’s “CME in Support of MOC” program in Section 3 of the Royal College’s MOC Program.

MIPS Improvement Activity:
This activity counts towards MIPS Improvement Activity requirements under the Medicare Access and CHIP Reauthorization Act of 2015 (MACRA). Clinicians should submit their improvement activities by attestation via the CMS Quality Payment Program website.

It is the policy of CME Outfitters, LLC, to ensure independence, balance, objectivity, and scientific rigor and integrity in all of their CE activities. Faculty must disclose to the participants any relationships with commercial companies whose products or devices may be mentioned in faculty presentations, or with the commercial supporter of this CE activity. CME Outfitters, LLC, has evaluated, identified, and mitigated any potential conflicts of interest through a rigorous content validation procedure, use of evidence-based data/research, and a multidisciplinary peer review process. The following information is for participant information only. It is not assumed that these relationships will have a negative impact on the presentations.

Dr. Roshan Lal reports the following financial relationships:

Advisory Board: Takeda Pharmaceuticals U.S.A., Inc. (Gaucher Advisory Board)

Consultant: FDNA INC.

Speakers Bureau: BioMarin (PKU)

Dr. Heldermon reports the following financial relationships:

Advisory Board: Chiesi USA, Inc.; Orphazyme; REGENXBIO Inc.; and Sanofi

Consultant: Atheneum; Cardinal Health; Curio Science; Guidepoint Global; IntrinsiQ Specialty Solutions; Reckner Healthcare; Sage Research; Sanofi; Targeted Oncology; and Third Bridge Consulting

Stock Shareholder (ownership Interest): Lacerta Therapeutics

Dr. Krainc reports no financial relationships to disclose.

Dr. Weinreb reports the following financial relationships:

Advisory Board: AVROBIO, Inc. (Disease Monitoring Safety Board); Pfizer Inc. (Medical Advisory Board for the Taliglucerase alfa Gaucher Registry); Sanofi (Scientific Advisory Board for the International Collaborative Gaucher Group (ICGG) Gaucher Registry); and Takeda Pharmaceuticals U.S.A., Inc. (Medical Advisory Board for Gaucher Disease)

Consultant: Pfizer Inc. and Sanofi

Speakers Bureau: Sanofi

The following peer reviewer and CME Outfitters staff have no financial relationships:

  • Jeffrey Helfand, DO (peer reviewer)
  • Thomas Mitchell (planning committee)
  • Kellie Busby, PharmD (planning committee)
  • Susan H. Yarbrough, CHCP (planning committee)
  • Sandra Caballero, PharmD (planning committee)
  • Sharon Tordoff (planning committee)

  • Faculty of this CE activity may include discussions of products or devices that are not currently labeled for use by the FDA. The faculty have been informed of their responsibility to disclose to the audience if they will be discussing off-label or investigational uses (any uses not approved by the FDA) of products or devices.

    Questions about this activity? Call us at 877.CME.PROS (877.263.7767).


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