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

IFEATURED ARTICLES Navigating Diagnosis and Treatment for Primary Biliary Cholangitis: The Integral Role of the Patient Voice https://checkrare.com/navigating-diagnosis-and-treatment-for-primary-biliary-cholangitis/ Sonal Kumar, MD, Assistant Professor of Medicine, and Director of Clinical Hepatology at Weill Cornell Medical College in New York, NY discusses the integral role of the patient voice in navigating treatment decisions and determining an individualized care plan for people living with primary biliary cholangitis (PBC) that reflects their needs and goals. Thyroid Eye Disease: Overview, Diagnosis, and Current and Emerging Treatment Options https://checkrare.com/thyroid-eye-disease-overview-and-diagnosis/ Raymond Douglas, MD, PhD, a world-leading clinician and thought leader in thyroid eye disease (TED) who has been integral to developing therapeutics for the disease, provides an overview of TED, including diagnosis challenges and current and emerging treatments for this rare disease. FDA NEWS 2025 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2025-orphan-drugs-pdufa-dates-and-fda-approvals/ Rare diseases and orphan drugs are at the forefront of novel development and groundbreaking research. Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. FDA Approves Myasthenia Gravis Treatment Nipocalimab https://checkrare.com/fda-approves-myasthenia-gravis-treatment-nipocalimab/ The U.S. Food and Drug Administration (FDA) has approved Imaavy (nipocalimab) for the treatment of generalized myasthenia gravis (MG). The approval is for patients, 12 years of age and older, with anti-acetylcholine receptor (AChR) or anti-muscle-specific kinase (MuSK) positive antibodies. FDA Approves The First Treatment for Immunoglobulin G4-Related Disease https://checkrare.com/fda-approves-the-first-treatment-for-immunoglobulin-g4-related-disease/ The U.S. Food and Drug Administration (FDA) has approved Uplizna (inebilizumab-cdon) for the treatment of adults with immunoglobulin G4-related disease (IgG4-RD).