Mucopolysaccharidoses (MPS) are a group of genetic diseases characterized by a deficiency of lysosomal enzymes responsible for the hydrolysis of glycosaminoglycans (GAGs), whose manifold biological roles contribute to a variety of clinical manifestations in patients presenting with MPS. MPS has seven different subcategories, of which Hunter syndrome, an X-linked recessive genetic disease, is MPS II. The syndrome is characterized by a deficiency in iduronate 2-sulfatase, which results in relatively high levels of the GAGs heparan and dermatan sulfate, leading to physical signs similar to MPS I, with the addition of aggressive behavior and developmental delay. The major complications of Hunter syndrome have to do with its widespread and varied symptoms. The nature of this disease is that multiple organ systems are impacted simultaneously, considerably decreasing patient quality of life. The most common symptoms are facial disfigurements, hepatosplenomegaly, and skeletal joint stiffness, but presentation can fluctuate widely between patients.
This activity identifies the critical components of the management process and offer solutions to close gaps in diagnosis and care, with the ultimate goals of improvement of Hunter syndrome management, treatment adherence, and health and cost outcomes.
Pediatricians, neurologists, endocrinologists, and primary care physicians; physician assistants, nurse practitioners, and pharmacists; and any other HCPs with an interest in or who may clinically encounter patients with Hunter syndrome.
This program is supported by an educational grant from Takeda.
This activity is free of charge.
Release Date: March 24, 2022 -- Expiration Date: March 24, 2024
Faculty: Natalia Gomez-Ospina, MD, PhD
Introductory content: review of Hunter syndrome
· Epidemiology: statistics (global prevalence)
· MPS risk and causes
o Pre-natal testing
· Pathophysiology, clinical presentation, disease course, and complications
· Diagnosis and the impact of late diagnosis or misdiagnosis
o Urine GAG analysis
o Enzyme test
· Patient case(s)
· Enzyme replacement therapy, including clinical data
· Physical therapy: role in patient care
· Emerging treatment options
o Gene therapy
o Enzyme replacement therapy
· 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 #202281720
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ABIM MOC Credit Type: Medical Knowledge
ABP MOC Recognition Statement: Successful completion of this CME activity, which includes participation in the evaluation component, enables the participant to earn up to 1.0 MOC points in the American Board of Pediatrics's (ABP) Maintenance of Certification (MOC) program. Participants will earn MOC points equivalent to the amount of CME credits claimed for the activity. It is the CME activity provider's responsibility to submit participant completion information to ACCME for the purpose of granting ABP MOC credit.
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Disclosures of Educational Planners: Charles Turck, PharmD, BCPS, BCCCP, CEO of ScientiaCME, has no relevant financial disclosures.
All relevant financial relationships have been mitigated.
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Commercial Support Disclosure: This program is supported by an educational grant from Takeda.
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