This study investigates the effects of an investigational medication on adults with moderate-to-severe ulcerative colitis (UC). Ulcerative colitis is a long-term condition that causes inflammation and sores in the digestive tract, particularly affecting the lining of the large intestine and rectum. The purpose of this study is to evaluate how …
This study investigates the effects of an investigational medication on adults with moderate-to-severe atopic dermatitis (AD). Atopic dermatitis is a chronic skin condition that causes itchy and inflamed skin. This study is for adults aged 18 to 80 who have not had success with topical treatments or cannot use them. …
This study investigates the effects of investigational medications compared to a placebo on lung capacity in people with Interstitial Lung Disease (ILD) caused by Systemic Sclerosis (SSc). Systemic Sclerosis is a condition where the skin and other organs become hard and thick due to increased collagen. The study aims to …
This study investigates the effects of vaccination during pregnancy on mothers and their babies. It looks at the health outcomes for both the mothers and their newborns when the mothers receive a vaccine called an investigational vaccine during pregnancy. The study aims to understand how this vaccine affects both maternal …
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 effects of an investigational medication on infants with a condition called Infantile-Onset Pompe Disease (IOPD). IOPD is a rare genetic disorder where the body cannot break down certain complex sugars due to a missing or non-working enzyme. This study aims to understand how routine use of …
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 …
