Natural course of Fabry disease: changing pattern of causes of death in FOS - Fabry Outcome Survey

Abstract

Fabry disease is a rare X-linked lysosomal storage disorder characterised by severe multisystemic involvement that leads to major organ failure and premature death in affected men and women. Over the past 7 years, the Fabry Outcome Survey (FOS) has collected data on the natural history of Fabry disease, and the long-term efficacy and safety of enzyme-replacement therapy.

This paper provides an update on the first analysis of FOS data.

Keywords

• enzyme replacement therapy
• agalsidase-alpha
• clinical-manifestations
• imvolvement
• prevalence
• efficacy
• galactosidase