Pompe Disease is a rare condition that affects muscles and organs. This study looks at people with two types of Pompe disease: Late-onset Pompe disease (LOPD) and Infantile-onset Pompe disease (IOPD). The study is observational, which means researchers will watch and learn without changing any treatments. Both people who are getting treatment and those who are not can join. The goal is to see how safe and effective treatments are and how they affect patients' lives over time.
Who can join? People with a confirmed Pompe disease diagnosis. However, if you are in another clinical trial or special program for new treatments, you cannot join.
- This study is global, meaning it involves people from all over the world.
- It is an observational registry, so you don’t change your current treatment.
- There are no direct medical risks, only regular follow-ups.
If you have Pompe disease and want to help researchers understand it better, this study might be for you. You will be helping doctors learn more about how the disease and its treatments affect people over time.