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Gaucher disease is characterized by a deficiency of the lysosomal enzyme glucocerebrosidase, resulting in the accumulation of sphingolipids throughout the body but most manifesting prominently in the bones. One of its first complications is the chronic anemia and a persistent bleeding risk. Another is the hepatosplenomegaly, which may be a part of the initial clinical presentation, as may the anatomical abnormalities of bone deformities and stunted growth.
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, which 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.
Annual meetings of large, national, professional societies offer an opportunity for HCPs to get a first glimpse at study results that have the potential to impact practice as provide a forum for an exchange of ideas and practices between thought leaders and less distinguished practitioners. Nevertheless, many HCPs will be unable to attend these conferences, justifying the creation of CME that summarize the major findings presented at these major meetings.
HCPs including: MD specialists, pediatricians, primary care physicians; geneticists; physician assistants, nurse practitioners, nurses, and pharmacists; and any other HCPs with an interest in or who may clinically encounter patients with Lysosomal Storage Disorders.
Commercial Support Disclosure: This program is supported by educational grants from Sanofi Genzyme.
Learners may participate in this activity free of charge.
Release Date: August 27, 2020 -- Expiration Date: August 27, 2022
Faculty: Neil Weinreb, MD FACP
By the end of the session the participant will be able to:
ACCME Activity #201540530ACCREDITATION FOR THIS COURSE HAS EXPIRED. YOU MAY VIEW THE PROGRAM, BUT CME / CE IS NO LONGER AVAILABLE AND NO CERTIFICATE WILL BE ISSUED.
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Faculty Disclosure: Neil J. Weinreb, MD, FACP, Voluntary Associate Professor of Human Genetics and Medicine (Hematology), University of Miami Miller School of Medicine, is a Medical/Scientific advisory board member for Genzyme - Sanofi, Shire HGT and Pfizer for which he has received honoraria. Consulting for Genzyme-Sanofi, Pfizer; Research support from Genzyme-Sanofi, Shire HGT.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP has no relevant financial disclosures.
Commercial Support Disclosure: This program is supported by educational grants from Sanofi Genzyme
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Advances in the management of Fabry disease
Treatment strategies in Fabry disease
Fabry disease: Updates from recent research findings