Pompe disease is a genetic disorder caused by the buildup of a sugar molecule called glycogen in the body's cells. This study observes patients worldwide who have Pompe disease, both in children (infantile-onset) and adults (late-onset). Some patients are on approved treatments, while others are not. The goal is to understand how well treatments work over time, how safe they are, and how they affect patients' quality of life.
Participants must be diagnosed with Pompe disease through specific tests. However, those involved in other experimental treatment studies cannot join. The study will collect data on any bad side effects from treatments and track how patients feel and live with the disease.
- Study is observational; no new treatment given.
- Participants can be from anywhere in the world.
- Data helps improve future treatment and care.