The MPS I Registry

Help Physicians Better Understand MPS I Disease by Participating in the MPS I Registry

Are you looking for a way to contribute first-hand to a larger goal that may help you and other people living with MPS I disease? Ask your doctor about 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 (see “Will my information be kept private”). This may ultimately contribute to earlier diagnosis, earlier intervention, and better disease management for you and other people living with MPS I disease.

Who can participate?

All people who have MPS I disease are eligible to participate, regardless of whether they are on disease specific treatment. Your doctor is the one who will enroll you in the MPS I Registry. He or she will explain the program and will ask you to sign an authorization form to participate in the MPS I Registry. Your participation is completely voluntary. A patient may decline to participate or withdraw consent at any time and without affecting his/her medical treatment.

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 treatment. The MPS I Registry is designed ultimately to provide the MPS I community with information about the MPS I disease population around the world.

Data collected through the Registry and reports published using that data may be reported to regulatory authorities for a variety of purposes including information regarding the use of enzyme replacement therapy (ERT), obtaining label indications for ERT and any other appropriate regulatory purpose.

The MPS I Registry is a program sponsored by BioMarin/Genzyme and administered by Genzyme Corporation. The MPS I Registry is overseen by an independent group of physicians, the MPS I Board of Advisors, with experience in researching and treating 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?

You will receive the same care by your doctor regardless of participation in the MPS I Registry. At your regular doctor visits, the medical information your doctor collects (such as information on your liver, spleen, blood, bones, quality of life, etc.) will be submitted to the MPS I Registry.

There is no end date for the MPS I Registry. You can participate in the MPS I Registry for as long as you and your 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 I participate?

When you volunteer to participate in the MPS I Registry, your medical information is pooled with other participants' and is used to assist physicians in studying trends and addressing specific questions about treating MPS I disease. This information may contribute to better care for you and other people living with MPS I disease.

Will my information be kept private?

Information that your doctor submits to the MPS I Registry will reference you by patient number and initials, and not by name. Only your doctor will have access to both your name and patient number. Information submitted to the MPS I Registry will be maintained as confidential in accordance with applicable national privacy regulations and other state and local laws related to medical information.

How do I participate in the MPS I Registry?

Before you can participate in the MPS I Registry, your doctor must call the MPS I Registry to enroll as a participating physician. Once your doctor is enrolled, you will be asked to sign a form that authorizes your medical information to be released to the MPS I Registry.

Speak with your doctor about participating in the MPS I Registry. Contact the MPS I Registry's toll-free number at 800-745-4447, ext. 15500 for more information.

To learn more, visit MPSIRegistry.com.