Accreditation/Credit Designation

Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Physicians’ Education Resource®, LLC, designates this enduring material for a maximum of 1.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Physicians’ Education Resource®, LLC, is approved by the California Board of Registered Nursing, Provider #16669, for 1.5 Contact Hours.

Acknowledgment of Support

This activity is supported by an educational grant from Amicus Therapeutics, Inc.

Cases and Conversations: Patient-Minded Approaches to Managing Fabry Disease

Release Date: April 30, 2020
Expiration Date: April 30, 2021

Activity Overview

This virtual symposium will bring leading clinical experts focused on early recognition and accurate diagnosis of the lysosomal storage disorder (LSD) Fabry disease, with the goal of establishing early therapeutic intervention. There is an increasing need for non-physician primary care healthcare providers, who provide a significant portion of patient care in rare diseases, to be aware of and to proactively diagnose and manage a wide variety of LSDs, including Fabry disease. In this activity, the experts will review cases that highlight key pathophysiologic mechanisms and clinical features involved in the evolution of Fabry disease. During their discussion, the clinicians will describe indicators that aid in diagnosis. They will also share their knowledge and interpretation of genetic evaluation and clinical trial data for current and emerging therapies to discern the potential clinical impact on patients with Fabry disease.

Acknowledgement of Support

This activity is supported by an educational grant from Amicus Therapeutics, Inc.

Instructions for This Activity and Receiving Credit

  1. Complete the activity (including pre- and post-activity assessments).
  2. Answer the evaluation questions.
  3. Request credit using the drop-down menu.

You may immediately download your certificate.

Target Audience

This educational program is intended for geneticists, genetic counselors, pediatricians, endocrinologists, primary care physicians, nurses, nurse practitioners, physician assistants, and other healthcare professionals who are involved in the care of patients with Fabry disease.

Learning Objectives

Upon successful completion of this educational activity, you should be better prepared to:

  • Review the varied spectrum of phenotypic presentation and underlying genotypes in patients with Fabry disease
  • Discuss appropriate testing in the workup of patients with Fabry disease and confirmation of disease-causing GLA mutation
  • Evaluate the efficacy and safety of enzyme replacement therapy (ERT) and small-molecule chaperone therapies and the treatment selection for patients with specific Fabry disease genotypes
  • Apply clinical trial evidence and testing information to optimize the management of patients with Fabry disease

Faculty, Staff, and Planners’ Disclosures

In accordance with ACCME Guidelines, PER® has identified and resolved all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.


Dawn A. Laney, MS, CGC, CCRC
Dawn A. Laney, MS, CGC, CCRC
Assistant Professor and Genetic Counselor
Director, Emory Genetic Clinical Trial Center
LSDC Program Leader
Emory University School of Medicine
Atlanta, GA

Disclosures: Grant Research Support: Sanofi Genzyme for ISS; Consultant: Amicus Therapeutics, Inc., Sanofi Genzyme, Takeda/Shire, Protalix Biotherapeutics; Other: Research coordinator for industry sponsored studies from Amicus Therapeutics, Inc., Protalix Biotherapeutics, Sangamo Therapeutics, Inc., Sanofi Genzyme, and Takeda/Shire. Served on the Fabry Registry NA Board of Advisors Co-Founder, ThinkGenetic, Inc.

Damara Ortiz, MD, FAAP, FACMG
Damara Ortiz, MD, FAAP, FACMG
Assistant Professor of Pediatrics
Program Director, Medical Genetics Residency
Medical Director, Genetic Counselor Training Program
Medical Genetics/PKU Department
UPMC Children’s Hospital of Pittsburgh
Pittsburgh, PA

Disclosures: Grant Research Support: Amicus Therapeutics, Inc., Sanofi Genzyme; Consultant: Sanofi Genzyme.

Raphael Schiffmann, MD, MHSc, FAAN
Raphael Schiffmann, MD, MHSc, FAAN
Medical Director, Institute of Metabolic Disease
Baylor Scott & White Research Institute
Dallas, TX

Disclosures: Grant Research Support: Amicus Therapeutics, Inc., Idorsia, Protalix Biotherapeutics, Takeda; Consultant: Amicus Therapeutics, Inc., Protalix Biotherapeutics.

The staff Physicians' Education Resource®, LLC (PER®) have no relevant financial relationships with commercial interests to disclose.

Off-Label Disclosure and Disclaimer

This activity may or may not discuss investigational, unapproved, or off-label use of drugs. Learners are advised to consult prescribing information for any products discussed. The information provided in this accredited activity is for continuing education purposes only and is not meant to substitute for the independent clinical judgment of a healthcare professional relative to diagnostic, treatment, or management options for a specific patient’s medical condition. The opinions expressed in the content are solely those of the individual faculty members and do not reflect those of PER® or any company that provided commercial support for this activity.

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