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 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, leading to muscle weakness and other health issues. The study does not involve any experimental treatments. Instead, it focuses on …
This study investigates the use of electronic health record (EHR) data from TriNetX, a global health research network, to conduct research related to chlamydia. Chlamydia is a common sexually transmitted infection caused by the bacterium Chlamydia trachomatis. The study aims to determine if the TriNetX database is a suitable real-world …
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 FDA-approved 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 …
