by James Radke, PhD | Aug 1, 2025
Joni Rutter, PhD, Acting Director at the National Center for Advancing Translational Sciences (NCATS) and Annie Kennedy, Chief of Policy and Advocacy at the EveryLife Foundation discuss their organizations’ collective studies showing the direct and indirect...
by James Radke, PhD | Aug 1, 2025
Henry J. Kaminski, MD, Professor of Medicine at The George Washington University describes the value of being in the Rare Diseases Clinical Research Network (RDCRN). Dr. Kaminsky leads the Dr. Kaminski leads the Myasthenia Gravis Rare Disease Network (MGNET), a...
by James Radke, PhD | Jul 3, 2025
Stephanie Davis, MD, and Thomas Ferkol, MD, Professors of Medicine at University of North Carolina at Chapel Hill, discuss the value of being part of the Rare Diseases Clinical Research Network (RDCRN). Drs. Davis and Ferkol are co-leaders of the Genetic Disorders...
by James Radke, PhD | May 27, 2025
Sanjay Ahuja, MD, Pediatric Hematologist, Chief Medical and Informatics Officer at Innovative Hematology and the Indiana Hemophilia and Thrombosis Center (IHTC), discusses the U.S. Food and Drug Administration (FDA) approval of Qfitlia (fitusiran) for patients with...
by James Radke, PhD | Apr 28, 2025
Shellye Horowitz, a patient with hemophilia and advocate, discusses the impact of hemophilia on women and girls. Hemophilia Hemophilia is a bleeding disorder that slows the blood clotting process. People with this disorder experience prolonged bleeding...
