Patients & Families

Help Physicians Better Understand Gaucher Disease by Participating in the Gaucher Registry

Are you looking for a way to contribute first-hand to a larger goal that may help you and other people living with Gaucher disease? Ask your doctor about participating in the Gaucher Registry.

The International Collaborative Gaucher Group (ICGG) Gaucher Registry is the largest cooperative, observational registry on Gaucher disease. Many medical professionals agree that since Gaucher disease is rare, accurate and complete information on the disease is especially important. The Gaucher Registry was established in order to better understand the natural history and progression of Gaucher disease.

Through the Gaucher Registry, healthcare providers and disease specialists can share clinical information about Gaucher disease. This may ultimately contribute to earlier diagnosis, earlier intervention, and better disease management for you and other people living with Gaucher disease.

Who can participate?

All people who have Gaucher 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 Gaucher Registry. He or she will explain the program and will ask you to sign an authorization to participate in the Registry. Your participation is completely voluntary.

What does the Gaucher Registry do?

In addition to helping understand the natural history progression of Gaucher disease, the Gaucher Registry is designed to help physicians understand the long-term effects of treatment. The Gaucher Registry is designed ultimately to provide the Gaucher community with information about the Gaucher 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 Gaucher Registry is a program sponsored by Genzyme Corporation and is overseen by an independent group of physicians the ICGG Coordinators, with experience in researching and treating people with Gaucher disease. The ICGG Coordinators help maintain the scientific integrity of the Gaucher Registry.

How does it work?

You will receive the same care by your doctor regardless of participation in the Gaucher 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 Gaucher Registry.

There is no end date for the Gaucher Registry. You can participate in the Gaucher 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 Gaucher disease keeps growing.

Why should I participate?

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

Will my information be kept private?

Yes. Information that your doctor submits to the Gaucher 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 Gaucher 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 Gaucher Registry?

Before you can participate in the Gaucher Registry, your doctor must call the Gaucher 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 Gaucher Registry.

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