Newborn Screening Update Following RFK’s Removal of Advisory Committee ++++++++

CLINICAL PERSPECTIVES Newborn Screening Update Following RFK’s Removal of Advisory Committee https://checkrare.com/newborn-screening-update-following-rfks-removal-of-advisory-committee/ Dean Suhr, President and co-founder of the MLD Foundation, discusses newborn screening updates and plans for gaining recommended uniform screening panel (RUSP) approval. ATLG Versus ATG in Graft-Versus-Host Disease https://checkrare.com/atlg-versus-atg-in-graft-versus-host-disease/ A new study published in American Journal of Hematology compared two treatment options for Graft versus host disease (GVHD). Efficacy of Nipocalimab To Treat Myasthenia Gravis: Updates From Vivacity-MG3 Trial https://checkrare.com/efficacy-of-nipcalimab-to-treat-myasthenia-gravis-updates-from-vivacity-mg3-trial/ Sindhu Ramchandren, MD, Executive Medical Director, Neuroscience and Disease Cluster Lead for Neuroimmunology and Neuromuscular Disorders at Johnson & Johnson, discusses updated results from the Vivacity-MG3 clinical trial in patients with generalized myasthenia gravis (MG). Effect of Increased IgG Levels on Prognosis in Primary Biliary Cholangitis https://checkrare.com/effect-of-increased-igg-levels-on-prognosis-in-primary-biliary-cholangitis/ A recent study published in Liver International analyzed patients with primary biliary cholangitis (PBC) who presented with higher IgG levels at diagnosis. Safety and Efficacy of Zilucoplan to Treat Myasthenia Gravis: Results from the RAISE Trial https://checkrare.com/safety-and-efficacy-of-zilucoplan-to-treat-myasthenia-gravis-results-from-the-raise-trial/ Michael Weiss, MD, Department of Neurology, University of Washington Medical Center, discusses results from the RAISE clinical trial testing the safety and efficacy of zilucoplan in patients with myasthenia gravis (MG). New FDA-Approved C3 Glomerulopathy (C3G) Treatment Targets Underlying Cause of Disease https://checkrare.com/new-fda-approved-c3-glomerulopathy-c3g-treatment-targets-underlying-cause-of-disease/ Carla Nester, MD, Professor of Pediatrics-Nephrology at the University of Iowa, discusses the recent U.S. Food and Drug Administration (FDA) approval of iptacopan as the first and only treatment of adults with C3 glomerulopathy (C3G). Clinical Outcomes in Peripheral Ulcerative Keratitis https://checkrare.com/clinical-outcomes-in-peripheral-ulcerative-keratitis/ A recent study published in the American Journal of Ophthalmology observed clinical outcomes in patients with peripheral ulcerative keratitis (PUK). Positive Results From MINT Study of Uplizna for Myasthenia Gravis https://checkrare.com/positive-results-from-mint-study-of-uplizna-for-myasthenia-gravis/ Richard J. Nowak, MD, Director of the Myasthenia Gravis Clinic at Yale University, discusses positive data from the MINT clinical trial evaluating Uplizna (inebilizumab-cdon) in adults with generalized myasthenia gravis (gMG).