This study investigates the safety and efficacy of an investigational medication in patients aged 10 to 65 years with sickle-cell disease (SCD). The study consists of two parts: a double-blind period where neither the participants nor the researchers know who is receiving the investigational medication or a placebo, and an …
This study investigates the effects of an investigational medication in adults with relapsing forms of multiple sclerosis (MS). The purpose of the study is to compare the annualized relapse rate (ARR) of participants taking the investigational medication versus those taking a daily oral dose of another medication.Participants will undergo regular …
This study investigates the clinical outcomes of patients with Gaucher disease. The study is observational, meaning it collects data from patients receiving routine care without altering their treatment. It aims to enhance understanding of the disease's variability, progression, and natural history to aid in better therapeutic interventions.Participants will undergo clinical …
This study investigates Fabry disease and its impact on pregnancy outcomes. The purpose of this observational study is to track the clinical outcomes of patients with Fabry disease, including those who are pregnant, to better understand the disease's progression and variability.Participants will undergo routine clinical assessments and receive standard care …
This study investigates Pompe disease, focusing on muscle function impacts due to glycogen buildup. The purpose of this observational study is to track the natural history and outcomes of Pompe disease in patients worldwide, regardless of treatment status. An observational study involves collecting data by observing participants receiving routine care, …
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 …
