This study investigates the long-term safety and tolerability of an investigational medication in adults with multiple sclerosis (MS). Participants in this study have previously been part of earlier trials of the investigational medication.Participants will continue or start open-label treatment with the investigational medication, depending on their previous study involvement. Some …
This study investigates the treatment response and safety of two dose regimens of an investigational medication compared with treatment withdrawal in individuals aged 12 years and older with moderate-to-severe atopic dermatitis. The study aims to see if participants who responded to the investigational medication in earlier studies can maintain their …
This study investigates the effectiveness and safety of three investigational medications compared to a placebo in treating primary focal segmental glomerulosclerosis (FSGS) or primary minimal change disease (MCD). These kidney disorders can lead to nephrotic syndrome, characterized by high levels of protein in the urine (proteinuria). The purpose of this …
This study investigates the efficacy and safety of an investigational medication in patients with moderate to severe Crohn's disease. The study involves different doses of the investigational medication compared to a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine.Participants will …
This study investigates the safety and ability of a 21-valent pneumococcal conjugate vaccine, an investigational vaccine, to stimulate the production of antibodies in healthy infants. The study compares this investigational vaccine to a licensed 15-valent pneumococcal vaccine, given alongside routine pediatric vaccines.Participants in the study will receive either the investigational …
This study investigates the clinical outcomes of patients with Gaucher disease through an observational registry. The purpose of the registry is to gather data on how the disease progresses and affects patients over time, without altering their treatment plans. This helps in understanding the natural history of the disease and …
This study investigates Mucopolysaccharidosis I (MPS I), a rare genetic disorder. The purpose of this observational study is to collect data on the natural history and progression of MPS I and to observe clinical responses in patients receiving enzyme replacement therapy or other treatments.Participants in this study will have their …
This study investigates Pompe disease, a rare genetic disorder. The purpose of this observational study is to track the disease's natural history and outcomes in patients, both those receiving treatment and those who are not. An observational study is a type of research study where data is collected by observing …
This study investigates Fabry disease, a rare genetic disorder. The purpose of this study is to track the routine clinical outcomes for patients with Fabry disease, without altering their treatment. This is an observational study, meaning that data is collected by observing participants receiving routine care, without changing their treatment, …
This study investigates pregnancy outcomes in women with Pompe disease. The study is observational, meaning it involves monitoring and collecting data on pregnant women with Pompe disease, whether or not they are receiving enzyme replacement therapy (ERT) or other treatments. The aim is to gather information on pregnancy complications and …
