CureGN-Penn PCC

原发性肾小球疾病包括微小病变病(MCD)、局灶节段病 肾小球硬化(FSGS)、免疫球蛋白A肾病(IGAN)和膜性 肾病(MN)占终末期肾病的10%，与明显的 成人和儿童的发病率和死亡率。尽管存在巨大的疾病负担 与这些异质性疾病相关的疾病发病机制、自然病史、预测因素 临床结果和治疗反应的预测因素仍未完全确定。至 为了解决这些知识差距，CureGN财团组建了一个由70人组成的大型财团 临床研究站点、肾病学家、具有不同专业知识的科学家、受影响的患者和 倡导团体、生物制药行业、联邦资助机构和监管机构 经纪公司。CureGN正在进行一项前瞻性的纵向观察研究，成功地 登记和保留近2400名成人和儿童的种族和民族平衡队列 患有IgAN、FSGS、MN和MCD的患者。我们建议维持和加强CureGN 财团基础设施及其辅助研究计划，以加速患者相关 肾小球疾病研究。我们将继续我们的核心前瞻性、纵向观测 肾小球疾病患者的研究，通过招募替代策略招募额外的受试者 保持2400人的活跃队列，同时存储和管理高质量的临床数据和 生物材料。这项基础性工作是由一个完善的研究管理结构进行的 以及数据协调中心(Arbor Research)之间运行良好的协作 健康和密歇根大学合作)和四个参与的临床中心 (由宾夕法尼亚大学、哥伦比亚大学、北卡罗来纳大学、 和中西部儿科联盟)。CureGN位于多个域的连接点，这些域 合作克服障碍，改善对肾小球疾病患者的护理。通过ITS 现在有经验的科学工作组，它的出版委员会和辅助研究 基础设施，通过启用多种相关知识的现代分析方法 域名，通过与NephCure肾脏联合管理研究机会池 国际，通过不断改进学习工具，通过加强职业发展 活动，并召集患者和公私合作，以验证和 实施精准医治，CureGN创造的资源应该 加快改善对肾小球疾病患者的护理。

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.