Overview
The Fabry Registry is an ongoing, international multi-center, strictly observational program
that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of
treatment status. No experimental intervention is involved; patients in the Registry undergo
clinical assessments and receive care as determined by the patient's treating physician.
The primary objectives of the Registry are:
- To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease;
- To assist the Fabry 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 Fabry population as a whole;
- To evaluate the long-term safety and effectiveness of Fabrazyme
Fabry Pregnancy Sub-registry: This Sub-registry is a multicenter, international,
longitudinal, observational, and voluntary program designed to track pregnancy outcomes for
any pregnant woman enrolled in the Fabry Registry, regardless of whether she is receiving
disease-specific therapy (such as enzyme replacement therapy with agalsidase beta) and
irrespective of the commercial product with which she may be treated. Data from the
Sub-registry are also used to fulfill various global regulatory requirements, to support
product development/reimbursement, and for other research and non-research-related purposes.
No experimental intervention is given; thus a patient will undergo clinical assessments and
receive standard of care treatment as determined by the patient's physician. If a patient
consents to this Sub-registry, information about the patient's medical and obstetric history,
pregnancy, and birth will be collected, and, if a patient consents to data collection for her
infant, data on infant growth through month 36 postpartum will be collected.
Description
Eligibility