This study investigates cold agglutinin disease (CAD) and cold agglutinin syndrome (CAS). CAD and CAS are rare conditions where the immune system mistakenly attacks and destroys red blood cells, especially at lower temperatures. The purpose of this study is to collect data from patients with these conditions to better understand …
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 …
This study investigates the effects of receiving an investigational vaccine during pregnancy or shortly before. The focus is on understanding how this vaccination might affect mothers and their babies, both during pregnancy and after birth.Participants in the study will be monitored throughout the pregnancy and the first year of the …
This study investigates the safety of an investigational medication taken by women during pregnancy and breastfeeding. The study aims to understand any risks this medication may have on pregnancy, as well as any complications or side effects it may cause in the developing baby, newborn, and infant. The study will …
This study investigates acid sphingomyelinase deficiency (ASMD), a rare genetic disorder that affects the breakdown of certain fats in the body, leading to their accumulation and causing harm to various organs. The purpose of this study is to observe and collect data on participants with ASMD without altering their treatment …
This study investigates the real-world use of an investigational medication in patients with hemophilia A, a condition where the blood does not clot properly due to the lack of a certain protein. The purpose of this study is to observe how this medication is used in the United States and …
This study investigates the long-term effects of an investigational medication in patients with Stage 2 Type 1 Diabetes (T1D). Stage 2 T1D is an early stage of the disease where there are changes in blood sugar levels, but no clinical symptoms are present yet. If the disease progresses to Stage …
This study investigates the use of prophylaxis treatment in patients with moderate or severe hemophilia A. Hemophilia A is a genetic disorder where the blood does not clot properly due to low levels of a protein called factor VIII. This study is being conducted in patients aged 12 to 50 …
This study investigates chronic primary Immune Thrombocytopenia (ITP), a condition where the immune system mistakenly attacks and destroys platelets, which are cells that help blood clot. The purpose of this study is to collect data from adult patients with ITP, focusing on those who have had the condition for more …
This study investigates the use of an investigational medication in people with Type 1 diabetes, a condition where the body's immune system attacks the cells in the pancreas that produce insulin. Type 1 diabetes is a chronic disease where the body's defense system mistakenly attacks specific cells within the pancreas, …
