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 1.0 AMA PRA Category 1 Credit™. 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 1.0 Contact Hour.

Acknowledgment of Support

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

Community Practice Connection™ Cases and Conversations: Patient-Minded Approaches to Managing Fabry Disease

Release Date: May 27, 2020
Expiration Date: May 27, 2021

Activity Overview

Fabry disease is a rare, X-linked lysosomal storage disorder that results in deficient or absent α-galactosidase and lysosomal accumulation of globotriaosylceramide and related glycosphingolipids across a wide variety of cells and tissues. Because of the importance of early recognition and accurate diagnosis of Fabry disease, there is an increasing need for healthcare providers across many specialties to be aware of and to proactively diagnose and manage this disease.

This Community Practice Connection™ program provides an in-depth review of some of the key highlights from a recent live webcast, Cases and Conversations: Patient-Minded Approaches to Managing Fabry Disease, held in April 2020. This unique and engaging multimedia activity is ideal for community-based clinicians and focuses on the practical aspects of the diagnosis and management of this disease. The program is designed for those who did not attend the live meeting and to help reinforce learnings for those who did.

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 Trials Center
Lysosomal Storage Disease Center 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 Inc; Other: Research coordinator for industry sponsored studies from Amicus Therapeutics Inc, Protalix BioTherapeutics Inc, 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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