MPS II Research Highlights From the WORLDSymposium (Mucopolysaccharidosis type II; MPS II; Hunter syndrome)

This accredited CME activity, led by Barbara Burton, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine, highlights the latest research about Mucopolysaccharidosis type II (MPS II; Hunter syndrome) presented at WORLDSymposium 2022 and provides expert analysis of its clinical relevance for busy members of the care team to help them care for patients they may encounter with this rare condition. MPS II, also known as Hunter syndrome, is a rare, progressive lysosomal disease caused by deficient activity of iduronate-2-sulfatase, attributable to pathogenic variants of the iduronate-2-sulfatase gene (IDS). Course facial features and skeletal irregularities are the dominant symptoms of the periphery but of great concern is the central symptoms (cognitive decline, seizures) that occur in the more severe cases.

Current treatment options are limited to improving peripheral but not central aspects of this disease and numerous therapies are in development to address the central symptoms that afflict patients with severe MPS II. Data about those therapies were presented at WORLDSymposium 2022 and discussed by Dr. Burton.