This study investigates Mucopolysaccharidosis I (MPS I), a rare genetic disorder. The purpose of this observational study is to collect data on the natural history and progression of MPS I and to observe clinical responses in patients receiving enzyme replacement therapy or other treatments.Participants in this study will have their …
This study investigates Pompe disease, a rare genetic disorder. The purpose of this observational study is to track the disease's natural history and outcomes in patients, both those receiving treatment and those who are not. An observational study is a type of research study where data is collected by observing …
This study investigates Fabry disease, a rare genetic disorder. The purpose of this study is to track the routine clinical outcomes for patients with Fabry disease, without altering their treatment. This is an observational study, meaning that data is collected by observing participants receiving routine care, without changing their treatment, …
