This study investigates the outcomes of patients with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. MPS I is a condition where the body cannot properly break down certain sugar molecules, leading to various health problems. The purpose of this study is to collect data to understand the natural history …
This study investigates Pompe disease, a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen. The purpose of this study is to understand how Pompe disease progresses over time and how it varies among patients. This includes looking at patients who receive treatment …
This study investigates Fabry disease, a rare genetic condition that affects the body's ability to break down a specific type of fat. The purpose of this study is to track the health outcomes of people with Fabry disease over time, regardless of the treatment they receive. The study includes both …
This study investigates pediatric patients with moderate to severe atopic dermatitis (AD), a skin condition causing red, itchy, and inflamed skin. The purpose is to understand the characteristics of these patients, especially when their condition is not well-managed with topical treatments, or when such treatments are not suitable.Participants will be …
