International Fabry Disease Registry and Pregnancy Sub-registry Tracking patient outcomes and pregnancy results

What this Trial Is About

Researchers are tracking patients with Fabry disease through an ongoing international observational program called the Fabry Registry. This registry collects routine clinical outcomes for patients regardless of whether they are receiving treatment. The study aims to better understand the diseases variability, progression, and natural history, including in women who carry one copy of the gene, and to help improve patient care by developing monitoring recommendations and reporting outcomes. Additionally, the registry evaluates the long-term safety and effectiveness of Fabrazyme4, a treatment used in Fabry disease. The registry includes a special pregnancy sub-registry for women with Fabry disease who are pregnant or have been pregnant. This sub-registry observes pregnancy outcomes and infant growth up to 36 months after birth, collecting medical and obstetric history and treatment details. No experimental treatments are given participants continue to receive their usual care as determined by their physicians. Data from both registries support regulatory requirements and ongoing research. Participants undergo regular clinical assessments and receive standard care from their doctors throughout the study. The research team collects data on disease progression, treatment effectiveness, pregnancy outcomes, and infant development. The study is observational, meaning no study drugs or procedures are administered. The total participation can last up to 33 years, allowing for long-term monitoring of safety and outcomes related to Fabry disease and pregnancy.

Fabry Disease Registry & Pregnancy Sub-registry