Health Dictionary
Reliable, easy-to-read patient information on common medical conditions — written in plain language, free of jargon, and grounded in current evidence. Browse the most frequent diagnoses, understand what they mean for everyday life, and learn which steps and questions help you have a focused conversation with your doctor.
Hurler syndrome medical therapy
Introduction
Overview
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
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.
References
References
Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Aditya Govindavarjhulla, M.B.B.S. [2]