The MPS I Registry

Help physicians better understand MPS I Disease by participating in the MPS I Registry

The MPS I Registry is an ongoing, observational database on MPS I disease. Many medical professionals agree that since MPS I disease is rare, accurate and complete information on the disease is especially important. The MPS I Registry was established in order to better understand the natural history and progression of MPS I disease.

Through the MPS I Registry, healthcare providers and disease specialists can share clinical information about MPS I disease. This may help contribute to a better understanding of MPS I disease.

Who can participate?

All people who have MPS I disease are eligible to participate. Doctors can enroll patients who sign an authorization form to participate in the MPS I Registry. A patient may decline to participate or withdraw consent at any time.

What does the MPS I Registry do?

In addition to helping understand the natural history progression of MPS I disease, the MPS I Registry is designed to help physicians understand the long-term effects of MPS I. The MPS I Registry is designed ultimately to provide the MPS I community with information about the MPS I disease population around the world.

The MPS I Registry is a program sponsored by BioMarin/Genzyme and administered by Sanofi Genzyme. The MPS I Registry is overseen by an independent group of physicians, the MPS I Board of Advisors, with experience in researching and managing people with MPS I disease. The MPS I Board of Advisors help maintain the scientific integrity of the MPS I Registry.

How does it work?

Patients receive the same care from their doctor regardless of participation in the MPS I Registry. During regular doctor visits, the medical information they collect (such as information on the patient's liver, spleen, blood, bones, quality of life, etc.) would be submitted to the MPS I Registry.

There is no end date for the MPS I Registry. Patients can participate in the MPS I Registry for as long as they and their doctor would like to. Long-term participation in the Registry will help make sure that the knowledge about MPS I disease keeps growing.

Why should patients participate?

When patients participate in the MPS I Registry, their medical information is pooled with other participants' and is used to assist physicians in studying trends and addressing specific questions about MPS I disease. This information may contribute to a better understanding of MPS I disease.

Is patient information kept private?

Yes, patient information is maintained as confidential by the registry.

To Find out More about the MPS I Registry, visit

Did You Know?

Genetic counselors are health professionals who are trained to help families understand genetic disorders such as MPS I disease. A genetic counselor can help you determine if you are a carrier of a gene defect that causes MPS I and can provide valuable information and support for family planning.

To learn more, click here.