Creating Knowledge on Pompe disease
The Pompe Registry, sponsored by Genzyme, is a global resource dedicated to improving the understanding of the variability and progression of Pompe disease.
The Pompe Registry works with physicians to compile patient outcomes data from routine clinical practice to provide the medical community with resources to:
- Encourage collaboration and shared expertise between colleagues
- Support the clinical practice of evidence-based medicine
- Develop individualized care plans based on benchmark comparisons of similar patients
- Facilitate significant research publications
Pompe Registry Objectives
The Pompe Registry is an ongoing, observational database that tracks the natural history and outcomes of patients with Pompe disease. All Pompe disease patients are eligible for enrollment irrespective of their treatment status, and all physicians managing patients with Pompe disease are encouraged to participate in the Pompe Registry.
The Pompe Registry provides a repository that allows for the exchange of information and aggregate data to facilitate clinical decision making and data reports, and serves as a vehicle for collaborative studies.
The primary objectives of the Pompe Registry are:
- To enhance the understanding of the variability, progression, identification, and natural history of the key manifestations of Pompe disease;
- To assist the Pompe disease medical community with the development of recommendations for monitoring patients and reports on patient outcomes to help optimize patient care;
- To characterize and describe the Pompe disease population as a whole; and
- To evaluate the long-term effectiveness and safety of available treatment options and support measures, including enzyme replacement therapy (ERT).
Role of Participating Physicians
Participating physicians are requested to submit participating patients’ data on a regular basis. It is recommended that data be submitted to the Pompe Registry according to the Recommended Schedule of Assessments found in the Pompe Registry Protocol. Pompe Registry Staff are available upon request to assist with any data collection questions.
Benefits of Participation
The Pompe Registry has several informative features that offer unique benefits to participating physicians.
Physicians' contributions of patient data to the Pompe Registry database benefit all other Pompe Registry participants, since data are pooled to study trends or address specific questions. Participating physicians, are encouraged to submit queries for specific information from the database to facilitate the management of Pompe patients. The Registry allows physicians to:
- Request individualized patient reports and informative clinical summaries to help monitor their patient’s disease status
- Provide a repository for the exchange of aggregate clinical data among physicians to facilitate clinical decision-making
- Access information on current treatment guidelines and practice patterns
- Through the Pompe Registry, the Pompe community has access to important resources on a disease that affects a small patient population. Management guidelines and publications from the Pompe Registry may contribute to earlier diagnosis and intervention.
There are several Pompe Registry-specific reports available to participating physicians. These reports provide important clinical summaries of individual patients as well as analytical summaries of the overall Registry population. Please visit www.pomperegistry.com to learn more.
Confidentiality and patient privacy are of primary concern to the Pompe Registry. The Registry complies with applicable national privacy regulations and other state and local laws relating to medical information. All patient and physician information submitted to the Pompe Registry is maintained as confidential.
- Patients are referenced by Registry ID number only
- Patients must authorize release of their clinical data to the Registry
- No site-to-site data comparisons are made
Contact the Pompe Registry
For more information on the Pompe Registry, please visit the Registry website or use the following contact information.
In the United States and non-European countries:
500 Kendall Street
Cambridge, MA 02142
Email: contact us
Genzyme Europe BV
NL 1411 DD Naarden
Email: contact us