Rare Disease Focus: Kidney and Urinary Diseases +++++ ++ +

CLINICAL PERSPECTIVES Diagnosis and Treatment of IgA Nephropathy https://checkrare.com/diagnosis-and-treatment-of-iga-nephropathy/ Jai Radhakrishnan, MD, Nephrologist and Professor at Columbia University Medical Center, discusses diagnosis and treatment of IgA nephropathy (IgAN). Sophie’s Hope Foundation: A GSD1b Patient Advocacy Organization https://checkrare.com/sophies-hope-foundation-a-gsd1b-patient-advocacy-organization/ Jamas LaFreniere, Founder of Sophie’s Hope Foundation and CURE GSD1b, and Blair Stone-Schneider, Executive Director of Sophie’s Hope Foundation, discuss their patient advocacy organization and starting a natural history study for glycogen storage disease type 1b (GSD1b). Impact of Approval of Pegcetacoplan for Rare Kidney Diseases https://checkrare.com/impact-of-approval-of-pegcetacoplan-for-rare-kidney-diseases/ Carla Nester, MD, University of Iowa Stead Family Children’s Hospital and lead investigator of the VALIANT study, discusses the impact of the recent approval of pegcetacoplan (Empaveli) for patients with C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Symptom and Treatment Burden in Fabry Disease https://checkrare.com/symptom-and-treatment-burden-in-fabry-disease/ Jack Johnson, Co-Founder and Executive Director of FSIG, discusses symptom and treatment burden in Fabry disease. New Data on Pegcetacoplan in Patients With C3G and IC-MPGN https://checkrare.com/new-data-on-pegcetacoplan-in-patients-with-c3g-and-ic-mpgn/ Fadi Fakhouri, MD, PhD, Professor of Nephrology at CHUV Lausanne, Switzerland, discusses new data on pegcetacoplan in patients with C3 glomerulopathy (C3G) and immune complex-mediated membranoproliferative glomerulonephritis (IC-MPGN). FDA Approves Obinutuzumab for Patients With Lupus Nephritis https://checkrare.com/fda-approves-obinutuzumab-for-patients-with-lupus-nephritis/ The U.S. FDA has approved Gazyva/Gazyvaro (obinutuzumab) for the treatment of adults with active lupus nephritis who are receiving standard of care. FDA Approves Updated REMS for IgA Nephropathy Treatment https://checkrare.com/fda-approves-updated-rems-for-iga-nephropathy-treatment/ The U.S. Food and Drug Administration (FDA) has approved the updated Risk Evaluation and Mitigation Strategy (REMS) for Filspari (sparsentan) for the treatment of IgA nephropathy (IgAN). Complement-Mediated Kidney Disorders: A Case Series https://checkrare.com/learning/p-complement-mediated-kidney-disorders-a-case-series/ Complement-Mediated Kidney Disorders: A Case Series Howard Trachtman, MD, FASN, and Carla M. Nester, MD, MSA, FASN, utilizes three unique case studies to address clinical questions which explore the complexities involved in diagnosing, treating, and managing patients with various complement-mediated kidney disorders, such as C3 glomerulopathy and atypical hemolytic uremic syndrome (aHUS).