Spotlight on the Critical Issues in the Treatment of Fabry Disease

This webcast was recorded during a live symposium at the American Society of Human Genetics (ASHG) Annual Meeting in Orlando, FL, USA, on October 19, 2017. During the symposium, the faculty of leading KOLs in the field provided an overview of the clinical picture of Fabry disease and its natural history. They offered guidance on identifying the correlation between genotype and phenotype and how this may affect disease progression. They also discussed the current need for individualized patient care, and reviewed the emerging treatment landscape on the basis of the latest scientific data. They used interactive plenary sessions and selected patient case presentations to encourage the exchange of views and opinions on diagnosing and treating this disease.

TARGET AUDIENCE

This activity has been designed to meet the educational needs of health care professionals involved in the diagnosis, treatment, or management of patients with Fabry disease.

EDUCATIONAL OBJECTIVES

Upon completion of this activity, participants should be better able to:

  1. Recognize that Fabry Disease has two major phenotypes: classic and later-onset.
  2. Describe genotype–phenotype correlations and how these may affect disease progression.
  3. Recognize that there is large variation in disease symptoms in heterozygous females due to random X-chromosomal inactivation.
  4. Explain how an individualized approach to patient care is necessary due to genotype, phenotype, and sex.

FACULTY

Robert J. Desnick, MD, PhD, DSc (Hon)
Icahn School of Medicine at Mount Sinai
New York, NY, USA

Robert J. Hopkin, MD
Cincinnati Children’s Hospital Medical Center
Cincinnati, OH, USA

Dominique P. Germain, MD, PhD
French Reference Center for Fabry Disease and University of Versailles St Quentin en Yvelines
Montigny, France

Disclosure of Conflicts of Interest

It is the policy of the Elsevier Office of Continuing Medical Education that all faculty, instructors, planners, managers, and other individuals who are in a position to control the content of this activity to disclose any real or apparent conflicts of interest relating to the topics of this educational activity.

The faculty reported the following financial relationships or relationships to products or devices they or their spouse/life partner have with commercial interests related to the content of this CME activity:

FacultyRelationship Identified With:

Robert J. Desnick, MD, PhD, DSc (Hon)

Consultant/Advisor: Amicus Therapeutics, Inc.; Sangamo Therapeutics, Inc.; Sanofi Genzyme

Stock Ownership: Amicus Therapeutics, Inc. (self and son); Sangamo Therapeutics, Inc.

Grant/Research Support: Sangamo Therapeutics, Inc.; Sanofi Genzyme

Royalties/Patents: Sanofi Genzyme, Shire HGT

Robert J. Hopkin, MD

Speakers Bureau: Actelion Pharmaceuticals US, Inc.; Alexion; Amicus Therapeutics, Inc.; Protalix Ltd.; Sanofi Genzyme

Consultant/Advisor: Actelion Pharmaceuticals US, Inc.; Alexion; Amicus Therapeutics, Inc.; Sanofi Genzyme

Grant/Research Support: Protalix Ltd.; Sanofi Genzyme

Dominique P. Germain, MD, PhD

Consultant/Advisor: Amicus Therapeutics, Sanofi Genzyme, Shire

Speakers Bureau: Amicus, Sanofi Genzyme, Shire

Grant/Research Support: Amicus, Sanofi Genzyme, Shire

Non-faculty: Sandy Breslow; Alison Kemp; Bernard M. Abrams, MD; Joke Artmanni; Megan Cannon, PhD; Marijke Verhaaf; hereby state that neither they nor their spouse/life partner have had any financial relationships to products or devices with any commercials interest related to the content of this activity of any amount during the past 12 months.

Financial Support

This activity has been supported by an independent educational grant from Sanofi Genzyme.

Provider Information

Jointly provided by the Elsevier Office of Continuing Medical Education and Excerpta Medica.

CME Credit (Physicians)

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 the Elsevier Office of Continuing Medical Education and Excerpta Medica. The Elsevier Office of Continuing Medical Education is accredited by the ACCME to provide continuing medical education for physicians.

The Elsevier Office of Continuing Medical Education designates this enduring activity for a maximum of 1.5 AMA PRA Category 1 Credit(s)™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

CME INQUIRIES/SPECIAL NEEDS

For all CME inquiries or special needs, please contact elsevierCME@elsevier.com.


Disclosure of Unlabeled Use: This educational activity may contain discussion of published and/or investigational uses of agents that are not indicated by the FDA. The Elsevier Office of Continuing Medical Education, Excerpta Medica, and Sanofi Genzyme do not recommend the use of any agent outside of the labeled indications.

Disclaimer: Participants have an implied responsibility to use the newly acquired information to enhance patient outcomes and their own professional development. The information presented in this activity is not meant to serve as a guideline for patient management. Any procedures, medications, or other courses of diagnosis or treatment discussed or suggested in this activity should not be used by clinicians without evaluation of their patient’s conditions and possible contraindications on dangers in use, review of any applicable manufacturer’s product information, and comparison with recommendations of other authorities.

Course summary
Available credit: 
  • 1.50 AMA PRA Category 1 Credit(s)™
  • 1.50 Non-physician
Course opens: 
01/24/2018
Course expires: 
01/23/2019
Rating: 
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Available Credit

  • 1.50 AMA PRA Category 1 Credit(s)™
  • 1.50 Non-physician

Accreditation Period

Course opens: 
01/24/2018
Course expires: 
01/23/2019
Please login or register to take this course.