Disease Progression

Both life expectancy and level of disability vary significantly between patients with rapidly advancing disease and patients with slowly advancing MPS VI. Patients with rapidly advancing disease may be wheelchair bound before the age of 15 and succumb to heart failure during adolescence or early adulthood. Some patients with slowly advancing disease maintain
significant levels of physical functioning into their 30s, and may survive into their 50s or 60s.^1,2

		Clinical Spectrum of MPS VI. As evidenced by these photographs of four 9-to 12-year-olds with MPS VI, the rate of disease progression can vary widely.
Photo courtesy of The National MPS Society, Inc.

MPS VI advances at different rates in different patients Rapidly advancing disease appears early in life with marked signs and symptoms
While slowly advancing disease may not present with signs and symptoms until adolescence or later in life
Variability in clinical presentation creates a diagnostic challenge MPS VI presents with a broad range of clinical symptoms Initial symptoms may be subtle, especially in patients with slowly advancing disease Initial symptoms are often common pediatric complaints, such as recurrent otitis media, recurrent sinopulmonary infections, slow growth, and umbilical or inguinal hernia

1. Swiedler SJ, Beck M, Bajbouj M, et al. Thres hold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet. Forthcoming.
2. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. Vol 3. 8th ed. New York, NY: McGraw- Hill; 2001:3421-3452.