Present and emerging strategies in the medical management of Hunter syndrome
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Activity Description / Statement of Need:
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 accredited educational activity would identify the critical components of the management process and offer solutions to close gaps in diagnosis and care, with the ultimate goals being the improvement of Hunter’s disease 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.