Hurler Syndrome
Alternate Names : Alpha-L-Iduronate Deficiency, MPS I, Mucopolysaccharidosis Type I
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Hurler Syndrome Treatment
Enzyme replacement therapy is now possible for patients with a defect in the enzyme a-L-iduronidase. (This includes individuals with Hurler syndrome, but also Scheie and Hurler-Scheie syndromes.) One study examined the effects of this intravenous enzyme replacement in a group of individuals with Hurler syndrome and Scheie syndrome. Many positive benefits were seen, including improved joint mobility, growth (in children), and heart function. Few side effects were reported and the patients tolerated the medication well. How the enzyme replacement affects long-term neurologic function or life-span is still unknown.
Bone marrow transplantation can improve some of the symptoms of the disease. To prevent mental retardation, a bone marrow transplant probably needs to be performed at a very young age. Other treatments depend on the affected organ system.
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