Forgot Password?
Return to Course Listing

CME: Treatment strategies in Fabry disease

Activity Description / Statement of Need:

In this online, self-learning activity:

Fabry disease is characterized by a deficiency of the glycoside hydrolase enzyme alpha galactosidase A, resulting in the accumulation of the glycolipid globotriaosylceramide throughout the body, particularly prominently in the blood vessels. A defect in the enzyme alpha galactosidase A results in glycosphingolipid accumulation, ultimately leading to multi-organ dysfunction and the patient’s premature death. Early symptoms, which occur during childhood, involve pain and may include Raynaud phenomenon, paresthesias, and arthralgia in the extremities and proximal limbs, as well as impaired gastrointestinal emptying, resulting in abdominal pain, diarrhea, early satiety, postprandial bloating, nausea, and vomiting. In adulthood, the disease’s impact spreads beyond and begins to affect the cardiac and renal systems.

Podcast available at: Apple Podcasts or wherever you get your podcasts. 

Target Audience:

The following healthcare professionals: cardiologists, nephrologists, pediatricians, and primary care physicians; physician assistants, nurse practitioners, nurses, and pharmacists; and any other healthcare professionals with an interest in or who may clinically encounter patients with Fabry disease.


Commercial Support Disclosure: This program is supported by an educational grant from Sanofi Genzyme.

Learners may participate in this activity free of charge.


Release Date: September 04, 2019 -- Expiration Date: September 04, 2021

Faculty: William Wilcox, MD

Agenda

Introduction

Epidemiology

  • Current statistics
  • Risk and causes

Future complications Pathophysiology and diagnosis

  • Clinical features
    • Arthralgia, kidney involvement, cardiac complications, dermatological manifestations
  • X-linked recessive gene disease pathology
  • Diagnostic methods
    • Clinical presentation, enzyme assay, GLA gene analysis, kidney biopsy

Treatment

  • Present and emerging therapy and their benefits on long-term outcomes
  • Enzyme replacement therapy
  • Emerging therapies
    • Substrate reduction therapy
    • Oral pharmacological chaperone
  • Supportive care, including pain management
  • Patient case(s)

Summary, conclusions, and best practice recap

Learning Objectives

By the end of the session the participant will be able to:

  • Describe the importance of quick and accurate Fabry disease diagnosis and treatment.
  • Using established methods, determine likelihood of Fabry disease given a patient case.
  • Appropriately describe available therapies used for treatment of Fabry disease and explain current literature supporting use of those therapies.
  • Design and implement an appropriate therapeutic plan for treatment of Fabry disease.
  • Describe future therapies currently being investigated for the treatment of Fabry disease.

Accreditation

This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through ScientiaCME. ScientiaCME is accredited by the ACCME to provide continuing medical education for physicians.

 

Credit Designation: ScientiaCME designates this educational activity for a maximum of  1.00 AMA PRA Category 1 Credit(s)™ toward the AMA Physician's Recognition Award. Physicians should only claim credit commensurate with the extent of their participation in the activity.

PhysiciansFor maintenance of certification (MOC) credit, you must enter your board certification ID # and birth date correctly.  It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive MOC credit for this activity. Please note: not all activities on this site provide MOC credit. If this activity does not specify that it provides MOC credit in this section, then it does NOT provide MOC credit.

 

Pharmacists

ScientiaCME is accredited by the Accreditation Council for Pharmacy Education (ACPE) as a provider of continuing pharmaceutical education. This program is approved for 1.00 hours (0.1 CEUs) of continuing education.  Proof of participation will be posted to your NABP CPE profile within 4 to 6 weeks to participants who have successfully completed the post-test.   Participants must participate in the entire presentation and complete the course evaluation to receive continuing pharmacy education credit.  ACPE # 0574-0000-19-031-H01-P.  CPE credit effective 9/04/19 and expires 9/04/21. This is a Knowledge (K)-type activity.

PharmacistsYou must enter your NABP # and birth date correctly so that proof of participation can be posted to your NABP CPE profile. It is the learner's responsibility to provide this information completely and accurately at the completion of the activity. Without providing it, the learner will NOT receive CPE credit for this activity.

Nurse Practitioners (NPs): The American Academy of Nurse Practitioners accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME.  ScientiaCME will provide NPs who successfully complete each activity with a certificate of participation indicating that the activity was designated for AMA PRA Category 1 Credit(s)™.

Physician Assistants: The American Academy of Physician Assistants accepts AMA PRA Category 1 Credit(s)™ from organizations accredited by the ACCME.


Faculty Disclosure and Resolution of COI

As a provider of continuing medical education, it is the policy of ScientiaCME to ensure balance, independence, objectivity, and scientific rigor in all of its educational activities. In accordance with this policy, faculty and educational planners must disclose any significant relationships with commercial interests whose products or devices may be mentioned in faculty presentations, and any relationships with the commercial supporter of the activity. The intent of this disclosure is to provide the intended audience with information on which they can make their own judgments. Additionally, in the event a conflict of interest (COI) does exist, it is the policy of ScientiaCME to ensure that the COI is resolved in order to ensure the integrity of the CME activity. For this CME activity, any COI has been resolved thru content review ScientiaCME.

Faculty Disclosure:  

Dr. William Wilcox, MD, PhD, Department of Human Genetics and Pediatrics, Emory University, has received financial support as a consultant and/or for clinical study grant from Amicus, Alexion, Arexis, Protalix, Shire, Genzyme / Sanofi, and Biomarin

Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, President of ScientiaCME, has no relevant conflicts of interest to disclose.

Instructions

  • Read the learning objectives above
  • Take the Pre-Test (optional). Completion of the pre-test will help us evaluate the knowedge gained by participating in this CME activity.
  • View the online activity. You may view this is in more than one session, and may pause or repeat any portion of the presentation if you need to.
  • Take the post-test
  • Complete the activity evaluation and CME registration. A CE certificate will be emailed to you immediately.

Cultural and Linguistic Competence

System Requirements

PC
Windows 7 or above
Internet Explorer 8
*Adobe Acrobat Reader
MAC
Mac OS 10.2.8
Safari or Chrome or Firefox
*Adobe Acrobat Reader
Internet Explorer is not supported on the Macintosh

*Required to view Printable PDF Version


Perform Pre-Test (optional)

Please take a few minutes to participate in the optional pre-test. It will help us measure the knowledge gained by participating in this activity.


Additional Courses That Are Related To This Activity

Fabry disease: Updates from recent research findings

Gaucher disease: Updates from recent research findings

Treatment strategies in Hunter Syndrome

Treatment strategies in Gaucher disease