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

January is Thyroid Eye Disease Awareness Month Thyroid Eye Disease: Overview, Diagnosis, and Current and Emerging Treatment Options https://checkrare.com/thyroid-eye-disease-overview-and-diagnosis/ Thyroid eye disease (TED) is a chronic endocrine, autoimmune disease characterized by immune-mediated orbital inflammation that greatly impacts a person’s vision. How the TED Community Organization Helps Patients With Thyroid Eye Disease Take Their Lives Back https://checkrare.com/how-the-ted-community-organization-helps-patients-with-thyroid-eye-disease-take-their-lives-back/ Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses her personal journey with thyroid eye disease (TED) and why she started the TED Community Organization. RESOURCES Rare Disease Recognition and Awareness Days https://checkrare.com/rare-disease-awareness-days-and-recognition-page/ Stay informed with our comprehensive calendar of rare disease awareness days, highlighting key dates and resources. Priority Review Voucher Tracking https://checkrare.com/priority-review-voucher/ The Priority Review Voucher (PRV) program incentivizes the development of drugs for rare pediatric and tropical diseases. FDA NEWS 2026 Orphan Drugs: PDUFA Dates and FDA Approvals https://checkrare.com/2026-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 Uplizna for the Treatment of Generalized Myasthenia Gravis https://checkrare.com/fda-approves-uplizna-for-the-treatment-of-generalized-myasthenia-gravis/ The U.S. FDA has approved Uplizna (inebilizumab) for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) and anti-muscle specific tyrosine kinase (MuSK) antibody positive. FDA Approves Oral Prophylactic Therapy (berotralstat) for Pediatric Patients With Hereditary Angioedema https://checkrare.com/fda-approves-oral-prophylactic-therapy-berotralstat-for-pediatric-patients-with-hereditary-angioedema/ The U.S FDA has approved Orladeyo (berotralstat) for prophylactic therapy in pediatric patients with hereditary angioedema (HAE) ages 2 to 12 years of age. FDA Approves Gene Therapy (Waskyra) for Patients with Wiskott-Aldrich Syndrome https://checkrare.com/fda-approves-gene-therapy-waskyra-for-patients-with-wiskott-aldrich-syndrome/ The U.S. FDA has approved Waskyra (etuvetidigene autotemcel) for the treatment of patients ages 6 months and older with Wiskott-Aldrich syndrome (WAS). FDA Approves Voyxact for Primary Immunoglobulin A Nephropathy https://checkrare.com/fda-approves-voyxact-for-primary-immunoglobulin-a-nephropathy/ The U.S. FDA has approved Voyxact (sibeprenlimab) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. FEATURED ARTICLE A New Test for Patient-Reporting of Mastocytosis Control https://checkrare.com/a-new-test-for-patient-reporting-of-mastocytosis-control/ A team of allergists, immunologists, and dermatologists sought to test a new patient-reported outcomes measure for assessing mastocytosis disease control. Their newly developed tool, called the Mastocytosis Control Test (MCT), was generated through interviews with adult patients with nonadvanced indolent systemic mastocytosis. CLINICAL PERSPECTIVES Long-Term Data With Dawnzera for the Treatment of Hereditary Angioedema https://checkrare.com/long-term-data-with-dawnzera-for-the-treatment-of-hereditary-angioedema/ Michael Manning, MD, Allergist and Immunologist, discusses long-term data with Dawnzera (donidalorsen) for the treatment of patients with hereditary angioedema (HAE). Advancing Thyroid Eye Disease Care: Integrating Tepezza, New Technology, and Optimizing Multidisciplinary Teams https://checkrare.com/advancing-thyroid-eye-disease-care-integrating-tepezza-new-technology-and-optimizing-multidisciplinary-teams/ Maja Kostic, MD, PhD, neuro-ophthalmologist at Bascom Palmer Eye Institute and Assistant Professor of Ophthalmology at the University of North Carolina at Chapel Hill, discusses advancing thyroid eye disease (TED) care. One-Year Phase 3 Data Testing Empaveli (pegcetacoplan) in Patients with C3G and IC-MPGN https://checkrare.com/one-year-phase-3-data-testing-empaveli-pegcetacoplan-in-patients-with-c3g-and-ic-mpgn/ Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses one-year phase 3 data of Empaveli (pegcetacoplan) for treatment of C3 glomerulopathy (C3G) and immune complex membranoproliferative glomerulonephritis (IC-MPGN). Interim Results From Study of Mezagitamab for the Treatment of IgA Nephropathy https://checkrare.com/interim-results-from-study-of-mezagitamab-for-the-treatment-of-iga-nephropathy/ Jonathan Barratt, Mayer Professor of Renal Medicine at the University of Leicester, discusses interim results from a study testing mezagitamab for the treatment of immunoglobulin A (IgA) nephropathy. The Development of Nipocalimab for the Treatment of Sjögren’s Disease https://checkrare.com/the-development-of-nipocalimab-for-the-treatment-of-sjogrens-disease/ Ghaith Noaiseh, MD, Associate Professor of Allergy, Clinical Immunology, and Rheumatology at The University of Kansas Medical Center, discusses the development of nipocalimab for the treatment of Sjögren’s disease (SjD).