| ENCYCLOPEDIA INDEX |
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Injury Disease Nutrition Poison Symptoms Surgery Test |
| A B C D E F G H I J K L M N O P Q R S T U V W X Y Z |
Hurler syndrome |
| Overview Symptoms Treatment Prevention |
| Alternative Names: |
| Alpha-L-iduronate deficiency; Mucopolysaccharidosis type I; MPS I |
| Treatment: |
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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. |
| Support Groups: |
| The National MPS Society 45 Packard Drive Bangor, ME 04401 207-947-1445 www.mpssociety.org |
| Expectations (prognosis): |
| Hurler syndrome is a disease with a poor prognosis. Children with this disease have significant progressive physical and mental deficiencies. Death can occur in late childhood, early adolescence, or adulthood. |
| Complications: |
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| Calling your health care provider: |
| Call your health care provider if you have a family history of Hurler syndrome and are considering having children, or if your child begins to develop a group of the characteristic signs and symptoms of Hurler syndrome. |
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