This study investigates the long-term safety and effectiveness of therapies for non-neoplastic hematologic disorders. The purpose is to collect data from participants with various blood disorders, such as hemophilia, von Willebrand Disease, and congenital platelet disorders, over a period of at least 15 years. Participants will be grouped into specific …
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 pregnancy outcomes in women with Pompe disease. This study is part of a larger registry that tracks the health of women with Pompe disease during pregnancy, whether or not they are receiving specific treatments like enzyme replacement therapy (ERT).Participants will undergo clinical assessments and receive standard 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 …
This study investigates the use of electronic health record (EHR) data from the TriNetX network to support chlamydia research. The purpose is to validate the use of diagnostic codes for identifying chlamydia infections and to describe testing and treatment patterns in the United States.Participants' medical records will be analyzed to …
This study investigates the effects of exposure to an investigational vaccine during pregnancy. The purpose is to assess maternal, obstetrical, pregnancy, neonatal, and infant outcomes among women who were vaccinated with the investigational vaccine during pregnancy or in the 30 days before their last menstrual period.Participants in this observational study …
This study investigates the long-term safety and immune response to an investigational therapy in children under 2 years old with a condition called Acid Sphingomyelinase Deficiency (ASMD). The purpose of this study is to observe how the investigational therapy affects children with this condition over time.Participants in the study will …
This study investigates Stage 2 Type 1 Diabetes (T1D), focusing on abnormal blood sugar control without symptoms. The study involves collecting general information on patients with Stage 2 T1D and observing the long-term effects of a specific investigational medication. Participants will be divided into two study arms: those exposed to …
