This study investigates pediatric patients with moderate to severe atopic dermatitis (AD), a skin condition that causes red, itchy rashes. The purpose is to understand the characteristics of these patients when their condition is not well-managed with creams or ointments, or when such treatments are not suitable. Participants will undergo …
This study investigates Gaucher disease, a genetic disorder where fatty substances accumulate in certain organs. The purpose is to understand the variability, progression, and natural history of the disease. Participants will be observed to gather data on their clinical outcomes, regardless of their treatment status. This information will help in …
This study investigates the clinical outcomes of patients with Fabry disease, a genetic disorder that affects the body's ability to break down a specific type of fat. The purpose is to understand the variability, progression, and natural history of the disease, including in females who carry one copy of the …
This study investigates the effects of an investigational vaccine on pregnant women and their babies. The purpose is to understand how the vaccine, given during pregnancy or shortly before, affects mothers and their newborns. The study will collect information from women who received the vaccine and follow them through their …
This study investigates the safety of an investigational medication when used by women during pregnancy and breastfeeding. It aims to understand the potential risks to both the mother and child, including any complications during pregnancy and effects on the developing baby. The study will collect data from women who have …
This study investigates Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disorder that affects the body's ability to break down certain fats. The purpose of this study is to observe children with ASMD types A, A/B, or B to gather information about the condition and its progression. ASMD can lead to …
This study investigates the real-world use of an investigational medication in patients with hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly due to a deficiency in a protein called factor VIII. The purpose of this study is to observe how patients in the US …
This study investigates Stage 2 Type 1 Diabetes (T1D), which is an early stage of T1D where blood sugar levels are abnormal but not yet causing symptoms. The purpose of this study is to gather information on patients with Stage 2 T1D and to learn more about the long-term effects …
