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Article: Researchers at Royal Manchester Children's Hospital release new data on Hunter syndrome.
- Article from:
- Gene Therapy Weekly
- Article date:
- September 11, 2008
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According to recent research published in the journal Genetics in Medicine, "Hunter syndrome (Mucopolysacchariclosis II) is a rare, X-linked disorder of glycosaminoglycan metabolism. It is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase, and in affected patients glycosaminoglycan accumulates in lysosomes of various tissues and organs and contributes to the pathophysiology of Hunter syndrome."
"The Hunter Outcome Survey (HOS) was established to better describe the natural history of this disorder and to evaluate the long-term effect of enzyme replacement therapy. HOS is. an international, multicenter, long-term observational survey that will ...