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 0.5 AMA PRA Category 1 Credits™. Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Acknowledgment of Support

This activity is supported by an educational grant from Chiesi USA.

BURST Clinical Consultations: Addressing Long-term Cardiac and Renal Outcomes in Fabry Disease

Release Date: November 30, 2021
Expiration Date: November 30, 2022

Activity Overview

Fabry disease (FD) is a rare, X-linked, lysosomal storage disease (LSD) resulting from pathogenic mutations in the alpha-galactosidase A gene (GLA). When α-GAL A is absent or deficient, glycosphingolipids accumulate in the lysosomes of a variety of cells, particularly in the small blood vessels, kidneys, heart, and nervous system. Substrate accumulation may also activate proinflammatory mechanisms which contribute to phenotypic expression. The leading cause of death in patients with FD is a cardiovascular (CV) event; in fact, many patients have the ‘cardiac variant’ which manifests primarily as left ventricular hypertrophy (LVH) or hypertrophic cardiomyopathy (HCM). Kidney disease is another major complication of FD, particularly in male patients. End-stage renal disease (ESRD) has been found to be the strongest indicator of CV disease progression. In this program, two specialists in caring for patients with Fabry disease discuss the management of patients with this highly variable disorder, including the benefits and limitations of enzyme replacement therapy and the need for multidisciplinary care to optimize quality of life for these patients.

Acknowledgement of Commercial Support

This activity is supported by an educational grant from Chiesi USA.

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 program is intended for cardiologists, nephrologists, gastroenterologists, geneticists, genetic counselors, and any other healthcare professionals involved in the management of Fabry disease.

Learning Objectives

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

  • Develop a differential diagnosis for patients presenting with symptoms consistent with Fabry disease
  • Analyze the efficacy and safety of current and emerging therapies in patients with Fabry disease
  • Apply knowledge of risk factors associated with cardiac and kidney disease progression to the long-term, multidisciplinary management of Fabry disease

Faculty, Staff, and Planners’ Disclosures

The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible companies.


Dawn Laney, MS, CGC, CCRC
Dawn Laney, MS, CGC, CCRC
Director, Genetic Clinical Research Center
Assistant Professor
Department of Human Genetics
Emory School of Medicine
Atlanta, GA

Disclosures: Grant/Research Support: Takeda, Sanofi Genzyme, Amicus Therapeutics, Protalix, Chiesi USA; Consultant: Spark, Sanofi, Amicus Therapeutics, Protalix, Chiesi USA; Stock/Shareholder: ThinkGenetic, Inc.


Manesh R. Patel, MD
Manesh R. Patel, MD
Division Chief
Department of Cardiology
Duke University Medical Center
Durham, NC

Disclosures: Grant/Research Support: Bayer, AstraZeneca, Janssen, HeartFlow, Procyrion; Consultant: Bayer, Janssen, Mytonomy, Procyrion.

PER® mitigated all COI for faculty, staff, and planners prior to the start of this activity by using a multistep process.

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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