Comprehensive Care for Patients with Gaucher Disease –
Diagnosing and Treating Gaucher in the Clinical Setting

Presented by the Johns Hopkins University School of Medicine.

Supported by educational grants from Actelion Pharmaceuticals US Inc, and Genzyme, a Sanofi Company.

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Release date: January 19, 2017

Valid through: January 19, 2019

Specialty: Neurology, Pediatrics

Media: Internet

Estimated Time to Complete Activity: 30 minutes

Target Audience

This activity is intended for hematologists/oncologists, primary care physicians, pediatricians, orthopedists, neurologists, cardiologists, pulmonologists, gastroenterologists, obstetrician/gynecologists, and geneticists. There are no prerequisites.

Program Overview

Optimal management of patients with Gaucher disease (GD) requires that clinicians provide accurate, timely diagnoses based on clinical manifestations and offer patients the most appropriate available treatments. As a supplement to the live meeting series, this interactive case-based activity will provide participants with the latest evidence regarding the diagnosis of patients with GD as well as clinical data on the efficacy and safety of current therapies, providing participants with the opportunity to explore clinical practice considerations from assessment and diagnosis, to treatment and monitoring to achieve long-term disease management.

Learning Objectives

After completing this activity, the participant will demonstrate the ability to:

  • DISCUSS the wide range of progressive clinical manifestations of GD in order to facilitate earlier recognition and diagnosis.
  • DESCRIBE current and emerging treatment options for GD, related safety and efficacy data, and their potential role in therapy.
  • IDENTIFY appropriate monitoring and considerations for the effective long-term management of patients with GD.

The Johns Hopkins University School of Medicine takes responsibility for the content, quality, and scientific integrity of this CME activity.

Credit Available

  • Physicians — maximum of 0.5 AMA PRA Category 1 Credit

All other healthcare professionals completing this course will be issued a statement of participation.

Steering Committee

Ada Hamosh, MD, MPH (Co-Chair)

Professor, Department of Pediatrics and Institute of Genetic Medicine
Professor, Department of Epidemiology,
Bloomberg School of Public Health
Johns Hopkins University School of Medicine

Pramod K. Mistry, MD, PhD, FRCP (Co-Chair)

Professor of Pediatrics and Internal Medicine
Chief, Department of Pediatric GI/Hepatology
Yale University School of Medicine

Neal J. Weinreb, MD, FACP

Voluntary Associate Professor of Human
Genetics and Medicine (Hematology)
University of Miami Miller School of Medicine
Co-Chair, Scientific Advisory Board of the International Collaborative Gaucher Group
Member, Medical Advisory Board of the National Gaucher Foundation

Physician Continuing Medical Education

Accreditation Statement

The Johns Hopkins University School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Credit Designation

The Johns Hopkins University School of Medicine designates this enduring material for a maximum of 0.5 AMA PRA Category 1 Credit™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

After reviewing this online activity, participants may receive a certificate by receiving a score of 70% or higher on the CME test and completing an evaluation. A certificate of participation will be available online immediately following successful completion of the activity.

Disclosure Policies

Full Disclosure Policy Affecting CME Activities
As a provider approved by the Accreditation Council for Continuing Medical Education (ACCME), Johns Hopkins University School of Medicine Office of Continuing Medical Education (OCME) requires attested and signed global disclosure of the existence of all financial interests or relationships with commercial interest from any individual in a position to control the content of a CME activity sponsored by OCME. The following relationships have been reported for this activity:

Planner
Ada Hamosh, MD, MPH, reports her spouse serving on a scientific advisory board for GlaxoSmithKline and as a consultant for Blade Therapeutics.

No other planners have indicated that they have any financial interests or relationships with a commercial entity.

Faculty
Pramod K. Mistry, MD, PHD, FRCP, reports receiving grants/research support and honoraria from and serving as a consultant for Genzyme, a Sanofi Company; receiving honoraria from and serving as a consultant for Shire; and serving as a consultant for Pfizer Inc.

No other faculty members have indicated that they have any financial interests or relationships with a commercial entity whose products or services are relevant to the content of their presentation(s).

Note: Grants to investigators at The Johns Hopkins University are negotiated and administered by the institution which receives the grants, typically through the Office of Research Administration. Individual investigators who participate in the sponsored project(s) are not directly compensated by the sponsor, but may receive salary or other support from the institution to support their effort on the project(s).

Instructions for Participation and Credit

The following is an interactive case simulation designed to help you gauge your basic knowledge of the topic and then direct you to areas you may need to focus on. It consists of 3 sections: an unaccredited pre-test, an interactive case study, and a CME post-test and evaluation. All 3 sections must be completed to receive CME credit. A certificate of participation will be available online immediately following successful completion of the activity.

Hardware and Software Requirements

Pentium 800 processor or greater, Windows 98/NT/200/XP or Mac OS 9/X or later, Microsoft Internet Explorer, Safari, Firefox, Windows Media Player 9.0 or later Flash player, 128 MB of RAM Monitor settings: High color at 800 x 600 pixels, Sound card and speakers, Adobe Acrobat Reader.

Disclosure of Unlabeled Use

No speaker has indicated that he/she will reference unlabeled/unapproved uses of drugs or products.

Disclaimer

The opinions and recommendations expressed by faculty and other experts whose input is included in this program are their own. This enduring material is produced for educational purposes only. Use of Johns Hopkins University School of Medicine name implies review of educational format design and approach. Please review the complete prescribing information of specific drugs or combination of drugs, including indications, contraindications, warnings and adverse effects before administering pharmacologic therapy to patients.

CONFIDENTIALITY DISCLAIMER FOR CME ACTIVITY PARTICIPANTS
I certify that I am attending a Johns Hopkins University School of Medicine CME activity for accredited training and/or educational purposes.

I understand that while I am attending in this capacity, I may be exposed to "protected health information," as that term is defined and used in Hopkins policies and in the federal HIPAA privacy regulations (the "Privacy Regulations"). Protected health information is information about a person's health or treatment that identifies the person. I pledge and agree to use and disclose any of this protected health information only for the training and/or educational purposes of my visit and to keep the information confidential. I agree not to post or discuss this protected health information, including pictures and/or videos, on any social media site (eg, Facebook, Twitter, etc), in any electronic messaging program or through any portable electronic device

I understand that I may direct to the Johns Hopkins Privacy Officer any questions I have about my obligations under this Confidentiality Pledge or under any of the Hopkins policies and procedures and applicable laws and regulations related to confidentiality. The contact information is: Johns Hopkins Privacy Officer, telephone: 410-735-6509, e-mail: HIPAA@jhmi.edu.

“The Office of Continuing Medical Education at the Johns Hopkins University School of Medicine, as provider of this activity, has relayed information with the CME attendees/participants and certifies that the visitor is attending for training, education and/or observation purposes only.”

For CME Questions, please contact the CME Office at 410-955-2959 or e-mail cmenet@jhmi.edu. For CME Certificates, please call 410-502-9634.


Johns Hopkins University School of Medicine Office of Continuing Medical Education
Turner 20/720 Rutland Avenue
Baltimore, Maryland
21205-2195

Reviewed & Approved by:
General Counsel, Johns Hopkins Medicine (4/1/03)
(Updated 4/09 and 3/14)

TABLE OF CONTENTS
Preassessment

Diagnosing and Treating Gaucher in the Clinical Setting

Pramod K. Mistry, MD, PhD, FRCP

Postassessment and Evaluation

This activity was developed in collaboration with ASiM.