martes, 12 de agosto de 2025

Rare Disease Focus: Autoimmune and Auto-Inflammatory Disorders +++++++

CLINICAL PERSPECTIVES Rilzabrutinib in the Treatment of IgG4-Related Diseases https://checkrare.com/rilzabrutinib-in-the-treatment-of-igg4-related-diseases/ John Stone, MD, Professor of Medicine at Harvard Medical School and Edward A. Fox Chair in Medicine at Massachusetts General Hospital, discusses data on rilzabrutinib in the treatment of IgG4-related disease (IgG4-RD). Recent Clinical Trial Data Testing Inebilizumab to Treat IgG4-Related Diseases https://checkrare.com/recent-clinical-trial-data-testing-inebilizumab-to-treat-igg4-related-diseases/ Matthias Löhr, MD, PhD, Professor of Gastroenterology & Hepatology at Karolinska Institutet, Stockholm, Sweden, discusses recent data from the MITIGATE clinical trial of inebilizumab for the treatment of IgG4-related disease (IgG4-RD). Effect of Elafibranor on Fatigue in Patients With PBC https://checkrare.com/effect-of-elafibranor-on-fatigue-in-patients-with-pbc/ Sandra Silvestri, MD, PhD, Chief Medical Officer at Ipsen, discusses the effect of elafibranor on fatigue in patients with primary biliary cholangitis (PBC). Unmet Needs of Patients With wAIHA https://checkrare.com/unmet-needs-of-patients-with-waiha/ Ann Leon, PharmD, Global Medical Affairs Leader of Benign Hematology at Johnson & Johnson, discusses the unmet needs of patients with warm autoimmune hemolytic anemia (wAIHA). Promising Results for Avapritinib in Observational Trial of Patients With Indolent Systemic Mastocytosis https://checkrare.com/promising-results-for-avapritinib-in-observational-trial-of-patients-with-indolent-systemic-mastocytosis/ Researchers from Berlin, Germany and Beirut, Lebanon have confirmed in a prospective, observational study that patients with uncontrolled symptoms of indolent systemic mastocytosis (ISM) can improve disease control and their quality of life with the use of avapritinib. Mezagitamab in Patients With Immune Thrombocytopenia https://checkrare.com/mezagitamab-in-patients-with-immune-thrombocytopenia/ David Kuter, MD, DPhil, Director of Clinical Hematology at Massachusetts General Hospital, discusses recent results of a trial of mezagitamab in patients with immune thrombocytopenia (ITP). 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).

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