What is MPS I?


MPS I (Mucopolysaccharidosis type I) is a rare genetic disease that people are born with. It happens when the body cannot make enough of an enzyme called IDUA (alpha-L-iduronidase).

Without enough IDUA, certain long-chain sugar molecules called GAGs (glycosaminoglycans) build up inside cells throughout the body. Over time, this buildup can damage organs, bones, joints, and other tissues.

MPS I can lead to serious health problems that affect movement, breathing, heart health, growth, and daily life. There is currently no cure.

What is IDUA, and why does it matter in MPS I?

IDUA (alpha-L-iduronidase) is an enzyme that helps the body break down and clear materials called glycosaminoglycans (GAGs), which can be toxic, in excess.
In people with MPS I, the body does not make enough working IDUA. Without it, GAGs build up over time and cause damage in many parts of the body, and can even result in early death.

Current and investigational treatments for MPS I are designed to help restore IDUA levels in the body.

Where can I find more information and resources about MPS I?

You are not alone. There are organizations dedicated to supporting MPS families with information, community, and resources.