This study investigates a registry platform for cancer patients, focusing on collecting data about their treatments, decisions, and outcomes. The aim is to understand the effectiveness and safety of anti-cancer therapies and supportive care. An observational study is a type of research study where data is collected by observing participants …
This study investigates atopic dermatitis (AD) in children, focusing on those with moderate to severe forms of the condition. Atopic dermatitis is a chronic skin condition characterized by itchy and inflamed skin. The study aims to understand the characteristics of these pediatric patients, especially when their condition is not well …
This study investigates the real-world effectiveness and treatment patterns of an investigational medication in adult patients diagnosed with prurigo nodularis (PN). Prurigo nodularis is a skin condition characterized by hard, itchy bumps. The purpose of this observational study is to understand how the investigational medication is used in managing PN …
This study investigates the effectiveness of an investigational medication against RSV, a virus that causes lung infections. The purpose is to see how well this medicine works in preventing RSV-related illnesses and medical visits in infants. RSV stands for Respiratory Syncytial Virus, which can lead to serious lung problems, especially …
This study investigates the long-term safety and efficacy of investigational gene therapy in individuals with severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Sickle cell disease is a condition where red blood cells become misshapen and can block blood flow, causing pain and other complications. Beta-thalassemia is a blood …
This study investigates how cannabis affects driving performance in adults who use it daily, occasionally, or have not recently used it. Researchers will use a driving simulator to measure the driving performance of these different groups. Cannabis is a plant that some people use for medical or recreational purposes. It …
This study investigates 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 observe the natural history and variability of Fabry disease in patients, including heterozygous females, without altering their treatment. An observational study …
This study investigates pregnancy outcomes in women with Pompe disease. Pompe disease is a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen due to a deficiency of the enzyme acid alpha-glucosidase. This study aims to track pregnancy complications and infant growth in …
This study investigates Gaucher disease, a genetic disorder where fat-laden Gaucher cells build up in areas like the spleen, liver, and bone marrow. The purpose of this observational study is to track the clinical outcomes of patients with Gaucher disease. An observational study is a type of research study where …
This study investigates the effects of an investigational medication in infants with Infantile-onset Pompe Disease (IOPD). Pompe Disease is a rare genetic disorder that affects the heart and muscles, caused by the buildup of glycogen in the body's cells. The purpose of this study is to observe clinical outcomes in …
