CLINICAL PERSPECTIVES: Recent Advances in Fabry Disease Clinical Research +++++

CLINICAL PERSPECTIVES Recent Advances in Fabry Disease Clinical Research David Warnock, MD, Professor of Medicine (Emeritus), University of Alabama at Birmingham, discusses recent advances in Fabry disease clinical research. Recent Advances in Fabry Disease Clinical Research By: Madaline Spencer| Published on: Jan 8, 2025 https://checkrare.com/recent-advances-in-fabry-disease-clinical-research/ GRIDS Symposium Celebrates its 10th Anniversary Ozlem Goker-Alpan, MD, Founder and Chief Medical Officer of the Lysosomal & Rare Disorders Research & Treatment Center (LDRTC), discusses her work in lysosomal storage diseases and the GRIDS conference. GRIDS Symposium Celebrates its 10th Anniversary By: Madaline Spencer| Published on: Jan 7, 2025 https://checkrare.com/grids-symposium-celebrates-its-10th-anniversary/ New Data on Investigational Dravet Syndrome Treatment Joseph E. Sullivan, MD, Professor of Neurology & Pediatrics at University of California San Francisco and Director of Benioff Children’s Hospital Pediatric Epilepsy Center of Excellence, discusses zovrevunersen for the treatment of patients with Dravet syndrome. New Data on Investigational Dravet Syndrome Treatment By: Madaline Spencer| Published on: Jan 6, 2025 https://checkrare.com/new-data-on-investigational-dravet-syndrome-treatment/ Citizen Health: AI-Powered Platform for Rare Conditions Farid Vij and Nasha Fitter, co-founders of Citizen Health, discuss how their organization helps rare disease patients. Citizen Health: AI-Powered Platform for Rare Conditions By: Madaline Spencer| Published on: Jan 3, 2025 https://checkrare.com/citizen-health-ai-powered-platform-for-rare-conditions/ PFIC and Alagille Syndrome Studies James Squires, MD, University of Pittsburgh, discusses recent data regarding Bylvay (odevixibat) in patients with progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome. PFIC and Alagille Syndrome Studies By: Madaline Spencer| Published on: Dec 30, 2024 https://checkrare.com/pfic-and-alagille-syndrome-studies/