Mucopolysaccharidosis I (MPS I) is a progressive, debilitating, and often life-threatening disease. It is an inherited disorder caused by a deficiency of alpha-L-iduronidase. This enzyme is required for the breakdown of bodily substances known as glycosaminoglycans (GAG). As GAG builds up in a person’s body, almost any organ can be damaged. Therefore, early diagnosis and treatment are important. MPS I is also known as Hurler syndrome, Hurler-Scheie syndrome, and Scheie syndrome.
Standard treatment is with Aldurazyme®
Aldurazyme is a registered trademark of Genzyme Corporation.