Primary Objectives: To describe the characteristics of pediatric patients with moderate to severe atopic dermatitis (AD) whose disease is not adequately controlled with topical therapies or when those therapies are not medically advisable. To evaluate the time-course of AD and selected atopic comorbidities. Secondary Objectives: To characterize disease burden and …
This study is a prospective, multinational, observational investigation designed to elucidate the real-world effectiveness and treatment patterns of dupilumab therapy in adult patients diagnosed with prurigo nodularis (PN). The primary objective of this study is to comprehensively characterize the real-world usage of dupilumab for the management of PN. In addition …
The primary objectives of the study are: To estimate the effectiveness of nirsevimab against polymerase chain reaction (PCR)-confirmed RSV (1) lower respiratory tract (LRTD) and (2) related medical encounters. the secondary objectives are: To estimate the effectiveness of nirsevimab against medical encounters with a respiratory related diagnosis. To estimate the …
Primary Objectives: -Long-term safety of BIVV003 in participants with severe sickle cell disease (SCD) and ST- 400 in participants with transfusion-dependent beta-thalassemia (TDT) Secondary Objectives: Long-term efficacy of the biological treatment effect of BIVV003 in SCD Long-term efficacy of the clinical treatment effect of BIVV003 on SCD-related clinical events Long-term …
Researchers at the University of Colorado Anschutz Medical Campus, Colorado School of Public Health, and Injury & Violence Prevention Center want to learn more about how people are affected by cannabis in different ways. We will use a driving simulator to compare the driving performance of adults who use cannabis …
The Pompe Registry is a global, multicenter, international, longitudinal, observational, and voluntary program for patients with Pompe disease, designed to track the disease's natural history and outcomes in patients, both treated and not. Data from the Registry are also used to fulfill various global regulatory commitments, to support product development/reimbursement, …
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary …
This Sub-registry is a multicenter, international, longitudinal, observational, and voluntary program designed to track pregnancy outcomes for any pregnant woman enrolled in the Pompe Registry, regardless of whether she is receiving disease-specific therapy (such as ERT with alglucosidase alfa or avalglucosidase alfa) and irrespective of the commercial product with which …
The ICGG Gaucher Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Gaucher disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The …
Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and …
