Pompe Disease is a rare illness that affects muscles and movement. This study is a Registry which means it collects information from patients over time. It’s international, meaning it includes people from all over the world. Patients with Pompe disease can join to help doctors learn more about how the disease changes and how well treatments work.
The study looks at both past and future data, which is called retrospective and prospective. To join, you must have a confirmed diagnosis of Pompe disease. This means tests show your body doesn't have enough of a certain enzyme, GAA, or there are changes in your GAA genes. There are no other restrictions to join.
- Participation is voluntary, which means you can choose to join or leave at any time.
- There is no specific treatment given in the study, just tracking of your health data.
- There is no financial compensation for participating, but your involvement can help improve future care for Pompe patients.