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.
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
Future complications Pathophysiology and diagnosis
Summary, conclusions, and best practice recap
By the end of the session the participant will be able to:
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Faculty Disclosure: 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.
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