In this online CME self-learning program:
Mucopolysaccaridoses (MPS) are group of genetic disease characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), whose manifold biological roles resulting in a variety of clinical manifestations in patients presenting with MPS. MPS has seven different subcategories, of which Hunter syndrome is MPS II. A deficiency in iduronate 2-sulfatase results in relatively high levels of the GAGs heparan and dermatan sulfate, resulting in physical signs similar to MPS I with the addition of aggressive behavior and developmental delay. Hunter syndrome is an X-linked recessive genetic disease, with males more likely to develop disease and females more likely to be carriers.
Agenda
Introduction |
Epidemiology
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Pathophysiology and diagnosis [Learning objective #1 and #2]
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Treatment [Learning objectives #1 and 3]
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Potential barriers to treatment and solutions [Learning objective #4]
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Summary, conclusions, and best practice recap |
The following healthcare professionals: pediatricians, neurologists, endocrinologists, 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 Hunter syndrome.
This program is supported by an educational grant from Shire
Release Date: June 29, 2017 -- Expiration Date: June 29, 2019
Faculty: Maria Escolar, MD
By the end of the session the participant will be able to:
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Faculty Disclosure: Maria Escolar, MDMS, Associate Professor of Pediatrics, Director, Program for the Study of Neurodevelopment in Rare Disorders, is Pittsburgh, PA is a consultant and/or has received research grants for Alexeon, Shire, Regenxbio, Retrofin, and Aveona.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP has no relevant conflicts of interest to disclose.
Commercial Support Disclosure: This program is supported by an educational grant from Shire
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