CureGN-Penn PCC

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Primary glomerular diseases including minimal change disease (MCD), focal segmental glomerulosclerosis (FSGS), immunoglobulin A nephropathy (IGAN), and membranous nephropathy (MN) account for 10% of end-stage renal disease and are associated with significant morbidity and mortality in both adults and children. Despite the significant disease burden associated with these heterogeneous disorders, disease pathogenesis, natural history, predictors of clinical outcomes and predictors of response to therapy remain incompletely defined. To address these gaps in knowledge, the CureGN consortium assembled a large consortium of 70 clinical study sites, nephrologists, scientists with diverse expertise, affected patients and advocacy groups, the biopharmaceutical industry, federal funding agencies, and regulatory agencies. CureGN is conducting a prospective, longitudinal observational study, successfully enrolling and retaining a race and ethnicity-balanced cohort of nearly 2400 adult and pediatric participants with IgAN, FSGS, MN and MCD. We propose to maintain and enhance the CureGN Consortium infrastructure and its ancillary study program to accelerate patient-relevant glomerular disease research. We will continue our core prospective, longitudinal observational study of glomerular disease patients, enrolling additional subjects by a recruit-to-replace strategy to maintain an active cohort of 2400, while banking and curating high quality clinical data and biomaterials. This foundational work is being conducted by a refined study administrative structure and a well-functioning collaboration between the Data Coordinating Center (at Arbor Research Collaborative for Health and the University of Michigan) and four Participating Clinical Centers (managed at the University of Pennsylvania, Columbia University, University of North Carolina, and the Midwest Pediatric Consortium). CureGN sits at the nexus of multiple domains that are collaborating in overcoming the barriers to improving care for glomerular disease patients. By its now experienced scientific working groups, its publications committee, and its ancillary study infrastructure, by enabling modern analytical approaches in multiple relatable knowledge domains, by administering a research opportunity pool in conjunction with NephCure Kidney International, by continuing refinement of study instruments, by enhancing career development activities, and by convening patient and public-private collaborations for validating and implementing precision medicine-based therapies, the resource created by CureGN should accelerate improving care of glomerular disease patients.