This study investigates the use of an investigational medication in patients with relapsed and/or refractory multiple myeloma (RRMM). Multiple myeloma is a type of cancer that affects plasma cells, which are found in the bone marrow. In this study, the effectiveness of the investigational medication will be assessed in terms …
This study investigates Mucopolysaccharidosis I (MPS I), a rare genetic disorder caused by the body's inability to produce a specific enzyme, leading to a buildup of complex sugars in the body. The purpose of this observational study is to collect data on patients with MPS I to understand the natural …
This study investigates the natural history and outcomes of Pompe disease, a rare genetic disorder caused by the buildup of a complex sugar called glycogen in the body's cells. The purpose of this study is to enhance understanding of the variability, progression, and identification of Pompe disease, while also characterizing …
This study investigates the effects of an investigational vaccine on maternal, obstetrical, pregnancy, and neonatal outcomes. It focuses on women vaccinated with the investigational vaccine during pregnancy or within 30 days before their last menstrual period (LMP). An observational study is a type of research study where data is collected …
This study investigates the safety of an investigational medication when used during pregnancy and breastfeeding. The purpose is to understand the risks associated with the medication on pregnancy outcomes, maternal health, and the development of the baby. The study will collect data on any complications or side effects that may …
This study investigates the impact of an investigational medication on long-term joint health in participants with Hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to a deficiency in a clotting factor called factor VIII. This can lead to excessive bleeding and joint damage.Participants …
This study investigates the long-term safety and immune response to a treatment in young children with Acid Sphingomyelinase Deficiency (ASMD). ASMD is a rare genetic disorder that affects the body's ability to break down certain fats, leading to harmful buildup in various organs. The purpose of this study is to …
