Aldurazyme® (laronidase)
MPS I is a progressive, debilitating and often life-threatening disease. It is an inherited disorder caused by a deficiency of an enzyme called alpha-L-iduronidase. This enzyme is required for the breakdown of certain substances in the body commonly referred to as GAG (glycosaminoglycans). As more and more GAG builds up in a person's body, almost all organs can be irreversibly damaged. Therefore, early diagnosis and treatment of MPS I is important. MPS I has also been called Hurler, Hurler-Scheie, and Scheie Syndromes.
Patient Consent & Agreement (PCA) Forms
Other Resources:
NORD - National Organization for Rare Diseases
Aldurazyme is a registered trademark of Genzyme Corporation.