by Madaline Spencer and James Radke, PhD | Mar 12, 2026
Brian Bigger, PhD, Professor of Advanced Therapeutics at the University of Edinburgh, discusses the first-in-human experience of autologous hematopoietic stem cell gene therapy (HSCGT) using a novel ex-vivo lentiviral (LV) platform to correct mucopolysaccharidosis II...
by Madaline Spencer and James Radke, PhD | Mar 11, 2026
Dawn Laney, MS, Genetic Counselor at Emory University School of Medicine, discusses the challenges and unmet needs of female patients with Fabry disease. Fabry disease is a type of lysosomal storage disease characterized by deficient alpha-galactosidase...
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...
