This study investigates the outcomes of pregnancies 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. The study includes women who are part of the Pompe Registry, whether or not they receive specific treatments …
The purpose of this study is to investigate cold agglutinin disease (CAD) and cold agglutinin syndrome (CAS). These conditions involve the immune system mistakenly attacking red blood cells at cold temperatures. The study will collect data on individuals with CAD or CAS to better understand these conditions.Participants in the study …
This study investigates pediatric patients with moderate to severe atopic dermatitis (AD), a skin condition causing red, itchy patches. The purpose is to understand the characteristics of these patients whose condition is not well-managed with creams or when such treatments are not suitable. It also looks at how AD and …
This study investigates the effects on mothers and their babies when exposed to an investigational vaccine during pregnancy or within 30 days before their last menstrual period (LMP). The study aims to gather data on pregnancy and infant outcomes by following women who received the vaccine and their children up …
This study investigates the effects of an investigational medication on women and their children during pregnancy and breastfeeding. The purpose is to understand any risks associated with the medication, such as complications during pregnancy or effects on the baby, including growth and development issues.Participants will provide data on their health …
This study investigates acid sphingomyelinase deficiency (ASMD), a rare genetic disorder that affects the body's ability to break down certain fats. This can lead to the buildup of harmful substances in various organs. The study is observational, meaning it does not involve any intervention or treatment changes, but collects data …
This study investigates the real-world use of an investigational medication in patients with hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to the lack of a clotting protein called factor VIII. The purpose of this study is to observe the effectiveness and safety …
This study investigates Stage 2 Type 1 Diabetes (T1D), which is an early stage of the disease where blood sugar levels are abnormal but not yet leading to symptoms. The purpose is to collect information on patients with Stage 2 T1D and observe the long-term effects of an investigational medication …
This study investigates the use of an investigational medication for patients with moderate or severe hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to low levels of a protein called factor VIII. The purpose of this study is to observe patients who are …
This study investigates chronic primary Immune Thrombocytopenia (ITP), a condition where the immune system mistakenly attacks and destroys platelets, which are necessary for normal blood clotting. The study aims to gather data from patient medical records and laboratory results to better understand this condition.Participants will have a routine blood test …
