Patients & Families

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

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

Many medical professionals agree that since Fabry disease is rare, gathering accurate and complete information on the disease is especially important. The Fabry Registry has been established in order to better understand the progression of Fabry disease, as well as the different ways in which Fabry disease affects men and women.

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

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

What does the Fabry Registry do?
In addition to helping understand Fabry disease progression in men and women, the Fabry Registry is designed to help physicians understand the long-term effects of treatment. The Fabry Registry will also monitor the effect of treatment on pregnant women and their offspring. The Fabry Registry is designed ultimately to provide the Fabry community with information about the Fabry 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 Fabry Registry is a program sponsored and administered by Genzyme Corporation and is overseen by an independent group of physicians with experience in researching and treating people with Fabry disease. This board of advisors helps maintain the scientific integrity of the Fabry Registry.

How does it work?
You will receive the same routine care by your doctor regardless of participation in the Fabry Registry. At your regular doctor visits, the medical information your doctor collects (such as information on your kidneys, heart, pain, hearing, quality of life, etc.) will be submitted to the Fabry Registry.

Unlike a clinical study, you can participate in the Fabry Registry for as long as you and your doctor would like. Long-term participation in the Fabry Registry will help make sure that the knowledge about Fabry disease keeps growing. The Fabry Registry program will proceed for a minimum of 15 years. Genzyme reserves the right to discontinue the Fabry Registry at any time.

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

Will my information be kept private?
Information that your doctor submits to the Fabry 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 Fabry 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 Fabry Registry?
Before you can participate in the Fabry Registry, your doctor must contact the Fabry 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 Fabry Registry.

Speak with your doctor about participating in the Fabry Registry. For more information about the Fabry Registry in the United States and non-European countries, please contact Genzyme (Cambridge, MA, USA) at 1-800-745-4447, ext. 15500. In Europe, please contact Genzyme (Naarden, The Netherlands) at +31-35-699-1232.