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Cardiology
Cardiac effects of enzyme replacement therapy for mucopolysaccharidosis probed
October 5th, 2006
A study from the United States has reported on an examination of cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I. "Mucopolysaccharidosis type I is a lethal autosomal recessive storage disease caused by a deficiency of lysosomal alpha-L-iduronidase and the consequent systemic accumulation of glycosaminoglycan. Cardiomyopathy and valvar insufficiency occur as glycosaminoglycan accumulates in the myocardium, expands the spongiosa of cardiac valves, and proliferates within the myointima of the epicardial coronary arteries." "Congestive heart failure and death occur within the first decade of life in the most severe...
Source: Blood Weekly (2006-10-05)
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