This study investigates the effects of an investigational medication on adults with Immune Thrombocytopenia (ITP) who have not responded to first-line treatments. The purpose of this study is to assess how early immune modulation with the investigational medication can impact the condition in these patients.Participants in this study will receive …
This study investigates the safety and ability of an investigational pneumococcal vaccine to stimulate the immune system in children and adolescents with sickle cell disease. Participants will receive either the investigational vaccine or a licensed pneumococcal vaccine, and researchers will compare the immune response between the two groups.Participants in the …
This study investigates the safety and efficacy of a one-time intravitreal injection of an investigational medication for participants with Geographic Atrophy (GA) secondary to Age-related Macular Degeneration (AMD). This study aims to understand how the investigational medication works in treating this condition.Participants will receive a one-time injection directly into the …
This study investigates the administration methods of an investigational medication for adults with multiple sclerosis. The purpose of this study is to compare the effects of subcutaneous (under the skin) administration with intravenous (into the vein) administration of the investigational medication in participants with relapsing multiple sclerosis (RMS) and non-relapsing …
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 effects and outcomes of systemic treatments in patients with atopic dermatitis (AD). The purpose of this study is to understand the patient journey and assess the safety and clinical outcomes of these treatments in a real-world setting. Atopic dermatitis can vary in severity, and systemic treatments …
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 …
