Fabry Disease Study

This study looks at people with Fabry disease, a rare genetic disorder, to see how treatments work over time. The study especially focuses on a treatment called migalastat. It is observational, meaning researchers will watch and record what happens without giving any new treatments. About 450 people worldwide will join, including those who use migalastat, enzyme replacement therapy (ERT), or no treatment at all. Participants will be watched for up to 5 years.

Key Points:

• The study lasts for up to 5 years with regular check-ups.
• Participants will be at least 18 years old and can be on migalastat, ERT, or untreated.
• Participants must not be in another clinical trial at the time of joining.

Participants need to give their consent to be part of the study. They are also required to have certain kidney function levels, measured by eGFR, which checks how well the kidneys clean the blood. This study is specific to the United States, and requirements may vary by location.