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 effectiveness of investigational treatments for people with severe sickle cell disease (SCD) and transfusion-dependent beta-thalassemia (TDT). Sickle cell disease is a condition where red blood cells, which carry oxygen, become misshapen and can block blood flow. Beta-thalassemia is a blood disorder that reduces …
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 Pompe disease, a rare genetic disorder that affects the muscles and other tissues in the body. The purpose of this study is to understand how Pompe disease progresses over time in patients who are receiving treatment and in those who are not. The study will collect data …
This study investigates Fabry disease, a genetic condition that affects the body's ability to break down a specific type of fat due to a deficiency in the enzyme alpha-galactosidase (αGAL). The purpose of the study is to understand the variability, progression, and natural history of Fabry disease, including in females …
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. This study does not involve any experimental treatments. Instead, it looks at how pregnancies progress and what outcomes occur, whether …
This study investigates the clinical outcomes of patients with Gaucher disease, a rare genetic disorder. Gaucher disease is caused by a deficiency in an enzyme called β-glucocerebrosidase, which leads to the buildup of certain fats in the body. This study does not involve any experimental treatments. Instead, it collects information …
This study investigates the effects of an investigational medication on infants with Infantile-Onset Pompe Disease (IOPD). IOPD is a rare genetic disorder that affects the heart and muscles, often leading to serious health problems. The purpose of this study is to see how the investigational medication affects survival without the …
This study investigates the effectiveness of an investigational medication for patients with relapsed or refractory multiple myeloma (RRMM). Multiple myeloma is a type of cancer that affects plasma cells, which are a kind of white blood cell in the bone marrow. Relapsed or refractory means that the cancer has returned …
This study investigates Mucopolysaccharidosis I (MPS I), a rare genetic disorder. The purpose is to understand the natural history and progression of MPS I, as well as how patients respond to treatments like enzyme replacement therapy. MPS I is a condition where the body lacks an enzyme called alpha-L-iduronidase, leading …
