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.
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 Takeda/Shire.
Learners may participate in this activity free of charge.
Release Date: October 02, 2020 -- Expiration Date: October 02, 2022
Faculty: Natalia Gomez-Ospina, MD, PhD
Introduction content: cursory refresher and review of Hunter syndrome
· Epidemiology: Statistics (global prevalence)
· MPS risk and causes
· Pathophysiology, clinical presentation, and disease course
· Comorbid conditions
· Diagnostic methods
· Patient case(s)
· Enzyme replacement therapy, including clinical data
· Physical Therapy: Role in patient care
· Emerging treatment options
· Potential barriers to treatment and solutions, including patient access programs
· Patient case(s)
Summary, conclusions, and best practice recap
By the end of the session the participant will be able to:
ACCME Activity #201540546
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Faculty Disclosure: Natalia Gomez-Ospina, MD, PhD, Assistant Professor of Pediatrics, Stanford University, has recieved funding as a consultant for Codexis, Inc. and is on the scientific advisory board for Integral Medicines.
Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, CEO of ScientiaCME, has no relevant conflicts of interest to disclose.
Commercial Support Disclosure: This program is supported by an educational grant from Takeda/Shire.
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