This study investigates the safety and ability of an investigational pneumococcal conjugate vaccine to stimulate the immune system in infants. The study compares this investigational vaccine with a licensed pneumococcal vaccine. Infants aged approximately 2 months will receive these vaccines alongside routine pediatric vaccines to see how well their bodies …
This study investigates the clinical outcomes of patients with Gaucher disease, a genetic disorder where fatty substances build up in certain organs, particularly the spleen and liver. The study is observational, meaning it does not involve any experimental treatments. Instead, it aims to understand the variability, progression, and natural history …
This study investigates the outcomes of patients with Mucopolysaccharidosis I (MPS I), a rare genetic disorder. MPS I is a condition where the body cannot properly break down certain sugar molecules, leading to various health problems. The purpose of this study is to collect data to understand the natural history …
This study investigates Pompe disease, a rare genetic disorder that affects the body's ability to break down a complex sugar called glycogen. The purpose of this study is to understand how Pompe disease progresses over time and how it varies among patients. This includes looking at patients who receive treatment …
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 …
