Hurler syndrome medical therapy

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Aditya Govindavarjhulla, M.B.B.S. [2]

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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 treated with 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-Scheie syndrome that does not cause mental retardation.