This study investigates a treatment for relapsed or refractory multiple myeloma. The purpose is to assess the effectiveness of a subcutaneous investigational medication delivered through a special system, combined with two other medications, in adults who have had 1 to 3 previous treatments for this condition. Participants will be involved …
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 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 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 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 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 …
The purpose of this study is to investigate the clinical outcomes of individuals with Fabry disease. This study is observational, meaning it collects data without altering the participants' treatment, to better understand the variability, progression, and natural history of Fabry disease, including in women who carry the gene but may …
