Hurler Syndrome, Hurler-Scheie Syndrome, and Scheie Syndrome (Mucopolysaccharidosis Type I) Medication

Updated: Sep 28, 2018
  • Author: Germaine L Defendi, MD, MS, FAAP; Chief Editor: Maria Descartes, MD  more...
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Medication

Enzyme, Replacement Therapy

Class Summary

Replacing the deficient enzyme may improve symptoms and delay disease-induced complications.

Laronidase (Aldurazyme)

Indicated to treat MPS I (Hurler syndrome and Hurler-Scheie syndrome). Used to increase catabolism of GAG, which accumulates with MPS I. Treatment has been shown to improve walking capacity and pulmonary function. Laronidase is a polymorphic variant of the human enzyme a-L-iduronidase produced by recombinant DNA technology.