Hurler syndrome medical therapy

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.

• 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.

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