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.
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.
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.
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.
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.
Yes, patient information is maintained as confidential by the registry.
To Find out More about the MPS I Registry, visit www.registrynxt.com
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.
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