This study investigates Pompe disease, focusing on muscle function impacts due to glycogen buildup. The purpose of this observational study is to track the natural history and outcomes of Pompe disease in patients worldwide, regardless of treatment status. An observational study involves collecting data by observing participants receiving routine care, …
This study investigates Fabry disease, focusing on the clinical outcomes of individuals with the condition, including females who carry the gene, without altering their treatment.The study focuses on understanding the variability, progression, and natural history of Fabry disease. It also aims to evaluate the long-term safety and effectiveness of treatments …
