This study investigates a vaccine for the H5 strain of pandemic flu. The purpose is to test three different dose levels of the vaccine—low, medium, and high—against a placebo. A placebo is an inactive substance that looks like the investigational vaccine but does not contain any medicine. The study aims …
This study investigates the treatment patterns and effectiveness of an investigational medication in adults with prurigo nodularis, a skin condition characterized by itchy nodules. The purpose of this study is to understand how this medication is used in real-world settings and to gather information about the patients who receive it.Participants …
This study investigates Gaucher disease, a genetic disorder where fatty substances accumulate in certain organs. The purpose is to understand the disease's progression and characteristics. Participants will be observed over time to gather data on their health outcomes, without any experimental treatments involved.Participants will undergo regular clinical assessments as part …
This study investigates the effects of an investigational medication on infants with Infantile-Onset Pompe Disease (IOPD). IOPD is a genetic disorder that affects muscle function due to the buildup of glycogen in the body's cells. The purpose of this study is to understand how the investigational medication impacts survival without …
This study investigates the condition known as Mucopolysaccharidosis I (MPS I), a rare genetic disorder. The purpose of this study is to gather information on the natural history and progression of MPS I, as well as the clinical responses of patients receiving enzyme replacement therapy or other treatments. MPS I …
This study investigates Pompe disease, a rare genetic disorder that affects the body's ability to break down glycogen, a type of sugar stored in muscles. The purpose of this study is to understand how Pompe disease progresses in patients and to gather information on the outcomes of those who are …
This study investigates the clinical outcomes of patients with Fabry disease, a rare genetic disorder that affects the body's ability to break down a specific type of fat. The purpose of this study is to understand how the disease progresses and varies among patients, including women who carry the gene …
This study investigates pregnancy outcomes in women with Pompe disease. Pompe disease is a genetic disorder that affects the body's ability to break down a complex sugar called glycogen. The study includes women who are pregnant and are part of the Pompe Registry, regardless of whether they are receiving enzyme …
This study investigates cold agglutinin disease (CAD) and cold agglutinin syndrome (CAS). These are rare conditions where the immune system mistakenly attacks and destroys red blood cells at cold temperatures. The purpose of this study is to collect data from individuals diagnosed with CAD or CAS to better understand these …
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 …
