Hurler syndrome medical therapy
|
Hurler Syndrome Microchapters |
|
Diagnosis |
|---|
|
Treatment |
|
Case Studies |
|
Hurler syndrome medical therapy On the Web |
|
American Roentgen Ray Society Images of Hurler syndrome medical therapy |
|
Risk calculators and risk factors for Hurler syndrome medical therapy |
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Aditya Govindavarjhulla, M.B.B.S. [2]
Please help WikiDoc by adding more content here. It's easy! Click here to learn about editing.
Overview
Treatment of Hurler syndrome involves supplementing the body with the deficient enzyme so that it can break down the GAG's. Enzyme replacement therapy has proven useful in reducing non-neurological symptoms and pain.
Medical Therapy
Enzyme Replacement Therapy
- Patient is given the drug Laronidase, or Aldurazyme.
- This is is a polymorphic variant of Alpha-L-iduronidase (lysosomal enzyme), produced by recombinant DNA technology.
- This enzyme is required for the hydrolysis of terminal Alpha-L-iduronic acid residues of heparan and dermatan sulfate.
- Enzyme replacement therapy is useful in dealing with problems of growth, breathing, heart and joints.
- It is not proven effective in cases of neurological symptoms or mental development problems.
- It is a good option for children with Scheie syndrome or Hurler's/Scheie syndrome that does not cause mental retardation.