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 Fabry disease, focusing on the clinical outcomes of individuals with the condition, including females who carry the gene, without altering their treatment.The study focuses on understanding the variability, progression, and natural history of Fabry disease. It also aims to evaluate the long-term safety and effectiveness of treatments …
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 the use of electronic health records (EHR) to validate diagnostic codes for chlamydia infections. The purpose is to determine if the TriNetX database can be a reliable source for chlamydia-related research. The study aims to assess the accuracy of ICD-10-CM codes for identifying chlamydia cases and to …
This study investigates the experiences of individuals with stage 2 Type 1 diabetes in the United States. The study compares those who have received an infusion with an investigational medication and those who have not.Participants will provide information on their quality of life, diabetes-related anxiety, and how they manage their …
This study investigates atopic dermatitis (AD) in adolescents and adults who are starting or changing systemic treatments. The purpose of this study is to understand the needs of patients with AD and to learn more about their experiences with the condition.The study aims to evaluate the safety and outcomes of …
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 effects of atopic dermatitis (AD) on pregnancy outcomes. This observational study will compare the incidence of adverse pregnancy outcomes, such as miscarriage or stillbirth, and the prevalence of adverse infant outcomes, like major congenital malformations or being small for gestational age, in women with AD who …
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 safety of an investigational medication when used by women during pregnancy and lactation. The purpose is to collect data on the effects of the medication on pregnancy outcomes, maternal health, and the developing child. Outcomes in infants exposed to the medication will be assessed, including their …
