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Fabry Disease: Updates in Pathophysiology, Natural History, and Management for the Nephrologist

Fabry Disease: Updates in Pathophysiology, Natural History, and Management for the Nephrologist

Format

Meeting Coverage

Time to Complete

1 hour

Released

October 31, 2019

Expires

October 31, 2020
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Maximum Credits

1.00 / AMA PRA Category 1 CreditTM

Accredited Provider



Presented by the Johns Hopkins School of Medicine

Commercial Supporter

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

Program Description

Fabry disease (FD) is an X-linked disorder of glycosphingolipid catabolism, which leads to the gradual accumulation of the enzyme's principle substrate, globotriaosylceramide; this results in the progressive damage of renal epithelial cells, cardiac myocytes, vascular endothelium, and neurons of the dorsal root ganglia and autonomic nervous system. Diagnosing patients with FD is challenged by variable clinical presentation, lack of pathognomonic features, and considerable symptom overlap with more prevalent conditions. With advances in molecular biology, there is now a better understanding of FD, including its prevalence, mechanisms of inheritance, genotype-phenotype relationships, and the impact of the disease in female carriers. These discoveries, along with the advent of efficacious, disease-specific therapies offer the potential to significantly improve the identification and care of patients living with FD. This archived in-office rounding activity will examine advances in our understanding of FD pathophysiology and genetics, including unique considerations in heterozygous female patients. It will also review treatment options for patients with FD and discuss strategies for nephrologists for developing patient-specific management plans.

Intended Audience

This archived in-office rounding activity is intended for nephrologists involved in the assessment and/or management of patients with FD.

Educational Objectives

After participating in this activity, the participant will demonstrate the ability to:

  • RECOGNIZE the natural history and clinical manifestations of FD to facilitate timely recognition and differential diagnosis in males and females.
  • DEVELOP patient-specific FD management plans for pediatric, female, and adult populations that consider published recommendations, individual clinical factors, patient goals, and novel FD therapies.
The Johns Hopkins School of Medicine takes responsibility for the content, quality, and scientific integrity of this CME activity.

Credit

1.00

Type

AMA PRA Category 1 Credit(s)TM

Accreditation Statement

The Johns Hopkins School of Medicine is accredited by the Accreditation Council for Continuing Medical Education (ACCME) to provide continuing medical education for physicians.

Designation Statement

The Johns Hopkins School of Medicine designates this enduring material for a maximum of 1.00 AMA PRA Category 1 CreditTM . Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Policy on Presenter and Provider Disclosure

It is the policy of the Johns Hopkins School of Medicine that the presenter and provider globally disclose conflicts of interest. The Johns Hopkins School of Medicine OCME has established policies that will identify and resolve conflicts of interest prior to this educational activity. Detailed disclosure will be made prior to presentation of the education.

Full Disclosure Policy Affecting CME Activities

As a provider approved by the Accreditation Council for Continuing Medical Education (ACCME), Johns Hopkins School of Medicine Office of Continuing Medical Education (OCME) requires attested and signed disclosure of the existence of financial relationships with commercial interest from any individual in a position to control the content of a CME activity sponsored by OCME.

The following relationships have been reported for this activity:

Planners
Dr. Atta reports performing contracted research as a principal investigator for GlaxoSmithKline, Merck & Co, Inc., and Sentien Biotechnologies, Inc.; and for receiving fees for non-CE services from Amgen, Inc.


Ms. Laney reports receiving a consulting fee from Genzyme Corporation for serving on the North American Registries Board for Fabry Disease as a consultant/speaker, as a study coordinator, and principal investigator; she also reports receiving a consulting fee for serving as a study coordinator, principal investigator, and as a speaker from Amicus Therapeutics; she also reports being a co-founder of, and having ownership interest in, ThinkGenetic, Inc; and for receiving a consulting fee from Protalix Biotherapeutics for serving as a speaker/educational grants/study coordinator.

No other planners have indicated that they have any financial interests or relationships with a commercial entity.

Note: Grants to investigators at the Johns Hopkins University are negotiated and administered by the institution which receives the grants, typically through the Office of Research Administration. Individual investigators who participate in the sponsored project(s) are not directly compensated by the sponsor, but may receive salary or other support from the institution to support their effort on the project(s).

Faculty

Mohamed G. Atta, MD, MPH, FASN (Co-Chair)
Professor of Medicine
Division of Nephrology
Johns Hopkins School of Medicine
Baltimore, Maryland

Dawn A. Laney, MS, CGC, CCRC (Co-Chair)
Assistant Professor and Director, Emory Genetic Clinical Trials Center
Program Leader
Emory Lysosomal Storage Disease Center
Department of Human Genetics
Emory University
Decatur, Georgia

Off-Label Product Discussion

No faculty member has indicated that his/her presentation will include information on off-label products.

Disclaimer

The opinions and recommendations expressed by faculty and other experts whose input is included in this activity are their own. This enduring activity is produced for educational purposes only. Use of Johns Hopkins School of Medicine name implies review of educational format, design, and approach. Please review the complete prescribing information of specific drugs or combination of drugs, including indications, contraindications, warnings, and adverse effects before administering pharmacologic therapy to patients.

Confidentiality Disclaimer for CME Activity Participants

I certify that I am participating in this Johns Hopkins School of Medicine CME activity for accredited training and/or educational purposes.

I understand that while I am participating in this capacity, I may be exposed to "protected health information," as that term is defined and used in Hopkins policies and in the federal HIPAA privacy regulations (the “Privacy Regulations”). Protected health information is information about a person's health or treatment that identifies the person.

I pledge and agree to use and disclose any of this protected health information only for the training and/or educational purposes of my visit and to keep the information confidential. I agree not to post or discuss this protected health information, including pictures and/or videos, on any social media site (eg, Facebook, Twitter, etc.), in any electronic messaging program or through any portable electronic device.

I understand that I may direct to the Johns Hopkins Privacy Officer any questions I have about my obligations under this Confidentiality Pledge or under any of the Hopkins policies and procedures and applicable laws and regulations related to confidentiality. The contact information is: Johns Hopkins Privacy Officer, telephone: 410-735-6509, e-mail: HIPAA@jhmi.edu.

“The Office of Continuing Medical Education at the Johns Hopkins School of Medicine, as sponsor of this activity, has relayed information with the CME attendees/participants and certify that the visitor is here for training, education and/or observation purposes only.”

For CME questions, please contact the CME Office at 410-955-2959 or email cmenet@jhmi.edu.

Johns Hopkins School of Medicine
Office of Continuing Medical Education
720 Rutland Avenue/Turner 20
Baltimore, Maryland 21205-2195
Reviewed & Approved by: General Counsel, Johns Hopkins Medicine (4/1/03)
(Updated 4/09 and 3/14)


myCME privacy policy

Table of Contents

Overview of Fabry Disease
Dawn A. Laney, MS, CGC, CCRC

Presentation and Progression of Fabry Disease
Dawn A. Laney, MS, CGC, CCRC

Diagnosis of Fabry Disease
Dawn A. Laney, MS, CGC, CCRC

Current and Future Therapeutic Options
Mohamed G. Atta, MD, MPH, FASN

Guidelines on Fabry Disease Management: Adults and Pediatric
Mohamed G. Atta, MD, MPH, FASN

HARDWARE & SOFTWARE REQUIREMENTS

Pentium 800 processor or greater, Windows 98/NT/200/XP or Mac OS 9/X or later, Microsoft Internet Explorer, Safari, Firefox, Windows Media Player 9.0 or later Flash player, 128 MB of RAM Monitor settings: High color at 800 x 600 pixels, Sound card and speakers, Adobe Acrobat Reader.

Instructions

The following activity is an archived in-office rounding activity designed to help you gauge your basic knowledge of the topic and then direct you to areas you may need to focus on. In order to receive CME credit, you must complete the pre-assessment, watch and listen to the video presentation, and complete the post-assessment and evaluation. A certificate of participation will be available online immediately following successful completion of the activity. Your online certificate will be saved on myCME within your Profile/CME History, which you can access at any time.

If you have any questions relating to the accreditation of this activity, please contact cmenet@jhmi.edu.

SYSTEM REQUIREMENTS

Hardware and Software Requirements
  • A computer with an internet connection
  • Internet Browser: Internet Explorer 7.x or higher, Firefox 4.x or higher, Safari 2.x or higher, or any other W3C standards compliant browser
  • Additional Software: Adobe Flash Player and/or an HTML 5 capable browser maybe required for video or audio playback. PowerPoint or Adobe Acrobat Reader may occasionally be required
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