This study is for people with Fabry disease, a genetic disorder affecting how your body processes fats. It aims to understand how well different treatments work, focusing mainly on migalastat, a type of medicine. The study will include at least 450 people worldwide, with three groups: those treated with migalastat, those treated with enzyme replacement therapy (ERT), and those not yet treated. Participants will be observed for up to 5 years to check the effectiveness and safety of these treatments and their impact on quality of life.
- The study will last for 5 years, and participants will have regular follow-ups.
- Participants must be 18 or older, have specific genetic markers, and not be part of another trial.
- Participants must understand and agree to the study details.
If you have Fabry disease and are considering joining, talk to your doctor about whether this study is right for you. Participation involves regular check-ins and possibly changing or starting a new treatment for your condition.