Al Qatif

The Pompe Registry is a global, multicenter, international program that follows patients with Pompe disease over time. It is an observational and voluntary study designed to track the natural history and outcomes of Pompe disease in both treated and untreated patients. The registry aims to improve understanding of the disease's variability, progression, identification, and natural history, with the goal of guiding and assessing therapeutic interventions. It also supports the Pompe medical community in developing monitoring recommendations and reporting patient outcomes to optimize care. Additionally, the registry helps characterize the Pompe disease population and evaluates the long-term effectiveness of alglucosidase alfa. This study collects data retrospectively and prospectively from patients worldwide diagnosed with Pompe disease. It does not involve any specific interventions or treatments but gathers comprehensive clinical information over time. Data collection includes medical history, diagnosis details, treatment status, and other relevant health information to better understand the disease and patient experiences. Participants contribute data through regular updates that capture their disease progression and treatment outcomes. Researchers use this information to study how Pompe disease manifests and changes over time, with a maximum follow-up period of 30 years. The registry helps fulfill regulatory commitments, supports product development and reimbursement, and provides valuable information for research and patient care improvements.