BURST Clinical Consultations: Addressing Long-term Cardiac and Renal Outcomes in Fabry Disease
December 7, 2021
December 7, 2022
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This activity is provided by Physicians’ Education Resource®, LLC.
This activity is supported by an educational grant from Chiesi USA.
- Physicians — maximum of 0.5 AMA PRA Category 1 Credit(s)™
All other health care professionals completing this course will be issued a statement of participation.
This program is intended for cardiologists, nephrologists, gastroenterologists, geneticists, genetic counselors, and any other healthcare professionals involved in the management of Fabry disease.
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.
Upon completion of this activity, you should be better able 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
Dawn Laney, MS, CGC, CCRC
Director, Genetic Clinical Research Center
Department of Human Genetics
Emory School of Medicine
Manesh R. Patel, MD
Department of Cardiology
Duke University Medical Center
Physician Accreditation Statement
Physicians’ Education Resource®, LLC, is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.
Physician Credit Designation
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.
Disclosures of Conflicts of Interest
The staff of Physicians’ Education Resource®, LLC have no relevant financial relationships with ineligible entities.
- Dawn Laney, MS, CGC, CCRC
- 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
- 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.
Instructions for Participation and Credit
- Complete the activity (including pre- and post-activity assessments).
- Answer the evaluation questions.
- Request credit using the drop-down menu.
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Google Chrome 28.0+ for Windows, Mac OS, or Linux
Mozilla Firefox 23.0+ for Windows, Mac OS, or Linux
Safari 6.0+ for Mac OSX 10.7 and above
Supported Phones & Tablets:
Android 4.0.3 and above
iPhone/iPad with iOS 6.1 or above
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.