by Scott Harwood | Mar 4, 2025
Heather Lau, MD, Executive Director of Global Clinical Development at Ultragenyx, discusses positive results regarding gene therapy UX111 for treatment of patients with Sanfilippo syndrome type A (MPS IIIA). MPS IIIA is a severe, progressive disorder that...
by Scott Harwood | Mar 3, 2025
Carlo Antozzi, MD, discusses results from the phase 3 Vivacity-MG3 study of nipocalimab in antibody positive adults with generalized myasthenia gravis (MG). MG is a chronic autoimmune neuromuscular disease characterized by weakness of the skeletal muscles....
by Scott Harwood | Aug 9, 2024
Hideki Garren, MD, PhD, Chief Medical Officer for Prothena, discusses the pathophysiology of AL amyloidosis and the mechanism of action of investigational drug birtamimab. Amyloid light chain (AL) amyloidosis is a progressive plasma cell disorder in...
by Scott Harwood | Aug 6, 2024
Alaa Hamed, Global Head of Medical Affairs for Rare Diseases for Sanofi, discusses two clinical trials that reinforce positive data for hemophilia treatments. Hemophilia is a rare bleeding disorder that slows the blood clotting process. People with this...
by Scott Harwood | Jul 31, 2024
Fadi Fakhouri, MD, PhD, Professor of Nephrology at Lausanne University, discusses the results from a phase 2 clinical trial testing the safety and efficacy of pegcetacoplan in patients with C3G and IC-MPGN. Complement 3 glomerulopathy (C3G) and...
