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Challenges in Disease Management for Type 1 Gaucher Disease
Live Event Live Event

Challenges in Disease Management for Type 1 Gaucher Disease


This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Med-IQ and National Gaucher Foundation. Med-IQ is accredited by the ACCME to provide continuing medical education for physicians.

Med-IQ       Gaucher Foundation

Released:
10/28/18
Expires:
10/27/19

Maximum Credits:

4.5
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By clicking "Continue," you are confirming that you have reviewed the CME information and read, understood, and unconditionally agreed to the Privacy Notice and Terms of Use.
Released:
10/28/18

Expires:
10/27/19

Maximum Credits:
4.5
Live Event Live Event
By clicking "Continue," you are confirming that you have reviewed the CME information and read, understood, and unconditionally agreed to the Privacy Notice and Terms of Use.

Released:
10/28/18
Expires:
10/27/19


Maximum Credits:
4.5


Overview: This live meeting will improve clinician’s knowledge of the variable presentation of GD1 in children, adolescents and adults in order to facilitate earlier diagnosis and determine appropriate management. Clinicians will also discuss genetic testing and prenatal and newborn screening and review the importance of counseling patients with GD1 that are planning to start a family. Finally, expert faculty will discuss GD1 cases studies in children, adolescents, and adults.

CME Information:

Chair/Presenter
Neal J. Weinreb, MD
Voluntary Associate Professor of Clinical Genetics and Medicine (Hematology)
Miller School of Medicine, University of Miami
Miami, FL
 
Presenters
Pramod K. Mistry, MBBS, PhD, FRCP
Professor of Medicine, Pediatrics, and Cellular & Molecular Physiology
Department of Internal Medicine (Digestive Diseases)
Yale New Haven Hospital
New Haven, CT
 
Heather Lau, MD, MS
Director, Lysosomal Storage Disease Program; Associate Director, Division of Neurogenetics
Assistant Professor, Department of Neurology
New York University School of Medicine
New York, NY
 
Manisha Balwani, MD
Associate Professor, Genetics and Genomic Sciences
Icahn School of Medicine at Mount Sinai
Division of Medical Genetics
New York, NY

Debra-Lynn Day-Salvatore, MD, PhD, FAAP, FACMG
St. Petersburgh University Hospital
New Brunswick, NJ
 
Ozlem Goker-Alpan, MD
Lysosomal Treatment Unit
Fairfax, VA
 
Paige Kaplan, MBBCh
Professor Emerita of Pediatrics
Perelman School of Medicine, University of Pennsylvania
Children’s Hospital of Philadelphia
Philadelphia, PA
 
John D. Loike, PhD (Key Note Speaker)
Professor of Biology, Touro College and University System
Adjunct Professor, Columbia University College of Physicians and Surgeons and Columbia College
New York, NY

Ellen Sidransky, MD
Chief, Section on Molecular Neurogenetics
Medical Genetics Branch
National Human Genome Research Institute, NIH
Bethesda, MD
 
Saul Yanovich, MD (Panel Moderator)
Medical Liaison
National Gaucher Foundation
Rockville, MD

Activity Planners
Amy M. Blum
Chief Operating Officer
National Gauche Foundation
Rockville, MD
 
Samantha Gordon
CME Specialist
Med-IQ
Baltimore, MD
 
Kathryn Schaefer, MSN, RN, CPHRM
Senior Manager, Accreditation and Compliance
Med-IQ
East Lansing, MI

Series Overview/Statement of Need
Gaucher disease (GD) is one of the most common lysosomal storage disorders with an estimated incidence of 1:40,000-60,000 in the general population with the highest frequency seen among the Ashkenazi Jewish population (1:800).
 
The presentation of Gaucher’s disease (GD) is extremely variable, with type 1 (GD1) being the most prevalent. Patients may present as early as infancy to as late as the eighth decade of life. In addition, there is substantial variability in organ involvement and disease progression. In general, patients with GD1 can present with hepatomegaly, splenomegaly, anemia, thrombocytopenia, and bone disease.
 
Once a GD1 diagnosis is suspected, confirmation is done through genetic testing. Due to GD1’s high degree of clinical variability, it is difficult to predict disease progression and even more challenging to determine when to initiate treatment.  Therefore, GD1 management requires an individually tailored multidisciplinary evaluation and monitoring. It is also important to counsel patients who have GD or are carriers about family planning.
 
This CME accredited session will provide expert insight into GD1 management including diagnosis, monitoring GD1 progression, early treatment initiation, counseling patients on family planning and the importance of prenatal and newborn screening. Clinicians will improve their knowledge of the variable presentation of GD1 in children, adolescents and adults in order to facilitate earlier diagnosis and determine appropriate management.  Clinicians will understand that importance of genetic testing, prenatal and newborn screening.  Clinicians will recognize the importance of counseling patients with GD1 who are planning on starting a family.  Finally, expert faculty will discuss GD1 cases studies in children, adolescents, and adults.

Learning Objective
Upon completion, participants should be able to:

  • Review updates to the recommendations for screening for GD
  • Identify patients at risk of GD through genetic counseling
  • Determine appropriate timing of genetic testing for GD
  • Differentiate clinical symptoms of GD1 over other subtypes
  • Evaluate changes in symptoms over the course of GD1
  • List the diagnostic tools critical for identification of GD1 symptoms
  • Identify early and late symptoms associated with GD1
  • Understand the implications of early treatment intervention for GD1 in children and adults
  • Describe outcomes of the latest clinical trials on early treatment initiation for GD1 in children and adults
  • Determine appropriate timing as to when to initiate GD1 therapy based on disease progression
  • Discuss the importance of newborn screening for GD
  • Implement steps to discuss with patients who are planning on having a baby
  • Discuss key points of GD1 in pregnancy
  • Develop a plan for parents with a newborn diagnosed with GD1
  • Assess cases for children and adults with GD1

Target Audience
This activity is intended for physicians that care for patients with Gaucher disease in Gaucher specialized treatment centers or who are interested in learning about Gaucher disease Type 1, including primary care physicians, internists, gastroenterologists, neurologists, hematologists, hepatologists, geneticists, oncologists, orthopedists, pain specialists, cardiologists, pulmonologists, obstetricians/gynecologists, and pediatricians.
 
Joint Providership Statement
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education (ACCME) through the joint providership of Med-IQ and National Gaucher Foundation. Med-IQ is accredited by the ACCME to provide continuing medical education for physicians.

Designation Statements
Med-IQ designates this live activity for a maximum of 4.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Instructions to Receive Credit
To receive credit, read the introductory CME material, attend the meeting, and complete the evaluation and attestation online.

Participants must complete the evaluation and attestation within 60 days of participation.

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. 
  
Manisha Balwani, MD
Consulting fees/advisory boards: Sanofi Genzyme
 
Debra-Lynn Day-Salvatore, MD, PhD, FAAP, FACMG
Consulting fees/advisory boards: Sanofi Genzyme, Shire
Fees received for promotional/non-CME activities: Sanofi Genzyme, Shire
Contracted research: Sanofi Genzyme
 
Ozlem Goker-Alpan, MD, has indicated no real or apparent conflicts.
 
Paige Kaplan, MBBCh
Consulting fees/advisory boards: Aeglea BioTherapeutics, Inc., Pfizer, Inc., Sanofi Genzyme
Ownership interest Spouse (stocks/stock options – excluding mutual funds): Pfizer, Inc.
 
Heather Lau, MD
Consulting fees/advisory boards: Actelion Pharmaceuticals Ltd, BioMarin, Pfizer, Inc., Sanofi Genzyme, Shire, Ultragenyx Pharmaceutical Inc., Prevail Therapeutics
Fees received for promotional/non-CME activities: BioMarin
Contracted research: Amicus, BioMarin, Mallinckrodt Pharmaceuticals, Pfizer, Inc., Sanofi Genzyme, Shire, Ultragenyx Pharmaceutical Inc.
 
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
 
Ellen Sidransky, MD, has indicated no real or apparent conflicts.
 
Neal J. Weinreb, MD
Consulting fees/advisory boards: Aeglea BioTherapeutics, Inc., Alexion Pharmaceuticals, Pfizer, Inc., Sanofi Genzyme
Fees received for promotional/non-CME activities: Shire

The 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, please contact Med-IQ.
Call (toll-free) 866 858 7434 or email info@med-iq.com.
                     
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ADA Statement
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.
 
Disclaimer
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 Pfizer, Inc.

Copyright
© 2018 National Gaucher Foundation, Inc. All rights reserved.

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.

Need help? If you need to speak with a member of our team, please call (toll-free) 866 858 7434 or email info@med-iq.com. For technical assistance, please refer to our support manual.