WHAT IS MPS-1?

MPS-1 is a disease caused by a deficiency of the enzyme alpha-L-iduronidase and resulting in a build-up of complex carbohydrates (GAGs) in the lysosomes of cells. What results is a breakdown of the cellular, tissue and organ systems, leading to:
1. impaired cardiac and pulmonary function
2. delayed physical development
3. deformities of the skeleton and joints, with short stature
4. reduced physical endurance, and
5. impaired mental function (in moderate to severe cases)
Most patients with moderate to severe disease die before adulthood as a result of complications from the disease.