Fabry Disease Research Highlights
Released On
May 15, 2025
Expires On
May 15, 2026
Media Type
Internet
Completion Time
30 minutes
Specialty
Neurology, Nephrology, Cardiology, Dermatology, Gastroenterology, Ophthalmology
Topics
Fabry Disease
Jointly provided by American Academy of CME and CheckRare CE.
supported by an independent educational grant from Amicus Therapeutics, Inc. and Chiesi USA.
Credit Available
- Physicians - maximum of 0.50 AMA PRA Category 1 Credits™
All other healthcare professionals completing this course will be issued a statement of participation.
Target Audience
This activity has been designed to meet the educational needs of physicians specializing in neurology, nephrology, cardiology, gastroenterology, ophthalmology, and dermatology. Other members of the care team may also participate.
Program Overview
Fabry disease is an inherited lysosomal storage disease caused by mutations in the GLA gene, disrupting the function of the enzyme, α-galactosidase. This results in the accumulation of globotriaosylceramide (GL-3) and its deacylated form, globotriaosylsphingosine (lyso-GL-3), leading to progressive disruption of multiple organ systems.
There are currently three treatment options available for Fabry disease, including two enzyme replacement therapies, agalsidase beta and pegunigalsidase alfa, and a chaperone therapy, migalastat. There are also other treatments in development (e.g., gene therapy, other enzyme replacement therapies) and some that are available in other countries (e.g., agalsidase alfa).
Due to the small patient population and variability in Fabry disease severity, it is challenging to develop properly powered, placebo-controlled clinical trials. As such, data shared at conferences like WORLDSymposium 2025 are crucial for guiding best practices in this disease area. This program, led by Dr. Eric Wallace, provides a summary of clinically relevant data presented at WORLDSymposium 2025 that can enhance the care of patients with Fabry disease. This 30-minute CME program highlights clinically relevant research presented at WORLDSymposium 2025 and hosted by Dr. Eric Wallace, Professor of Medicine at University of Alabama Medical School.
Learning Objectives
After participating in the activity, learners should be better able to:
- Describe the latest research being presented to better manage individuals with Fabry disease and its clinical relevance.
Faculty
Eric Wallace, MD
Professor of Medicine
Department of Nephrology
University of Alabama Medical School
Accreditation Statement
In support of improving patient care, this activity has been planned and implemented by American Academy of CME, Inc. and CheckRare CE. American Academy of CME, Inc. is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.
Credit Designation
American Academy of CME designates this enduring material for a maximum of 0.50 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
American Academy of CME and CheckRare CE staff, have no relevant financial relationships with any ineligible companies.
This activity will review off-label or investigational information.
Faculty Educator
Dr. Wallace discloses the following relevant financial relationships with ineligible companies:
- Advisory Board Consultant: Sanofi-Genzyme, Chiesi, Kyowa Kirin, Sangamo, Natera
- Grant/Research Support: Sanofi-Genzyme, Chiesi, Uniqure, Idorsia, Amicus
Instructions for Participation and Credit
There are no fees for participating and receiving CME credit for this enduring activity. To receive CME credit participants must:
- Read the CME/CE information and faculty disclosures.
- Participate in the online activity.
- Submit the post-test (achieving a passing score of 70%) and the evaluation form.
Certificates will be immediately available for the participant.
Course Viewing Requirements
Supported Browsers:
Internet Explorer 8.0+ for Windows 2003, Vista, XP, Windows 7, Windows 8.1 and above
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
Disclosure of Unlabeled Use
American Academy of CME, Inc. requires that faculty participating in any CME activity disclose to the audience when discussing any unlabeled or investigational use of any commercial product or device not yet approved for use in the United States. This activity will review off-label or investigational information.
Disclaimer
The opinions expressed in this educational activity are those of the faculty, and do not represent those of the American Academy of CME or CheckRare CE. This activity is intended as a supplement to existing knowledge, published information, and practice guidelines. Learners should appraise the information presented critically, and draw conclusions only after careful consideration of all available scientific information.
Contact Information
For CME questions please contact: [email protected]