This study investigates the long-term safety and effects of an investigational medication in adults with Alpha-1 Antitrypsin Deficiency (AATD) emphysema. AATD is a genetic condition that can lead to lung diseases like emphysema. Emphysema is a lung condition that causes shortness of breath due to damaged air sacs in the …
This study investigates the safety and tolerability of an investigational medication combined with other agents in people with certain blood cancers that express a protein called CD123. Hematological malignancies are types of cancer that begin in blood-forming tissue, such as the bone marrow, or in the cells of the immune …
This study investigates the effect of an investigational medication compared to a placebo in people with recently diagnosed Type 1 diabetes who are on insulin therapy. Type 1 diabetes is a chronic disease where the body's defense system mistakenly attacks specific cells within the pancreas, which make insulin. Insulin helps …
This study investigates the safety and immune response of different investigational vaccines for respiratory viruses. The vaccines include formulations for trivalent influenza and a combined vaccine for respiratory syncytial virus, human metapneumovirus, and parainfluenza virus type 3. A lipid nanoparticle is used in the vaccines to help deliver the vaccine …
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 pediatric patients with moderate to severe atopic dermatitis (AD), a skin condition causing red, itchy, and inflamed skin. The purpose is to understand the characteristics of these patients, especially when their condition is not well-managed with topical treatments, or when such treatments are not suitable.Participants will be …
