by James Radke, PhD | Feb 16, 2022
Jerry Vockley, MD, PhD, Head of the Division of Medical Genetics at UPMC Children’s Hospital of Pittsburgh, discusses the phase 1/2 trial of the gene therapy, 4D-310, in Fabry disease. Data from this study was recently presented at WORLDSymposium 2022. Fabry...
by Peter Ciszewski | Feb 16, 2022
Paula Ragan, PhD, CEO and President of X4 Pharmaceuticals, describes the CXCR4 pathway, and how mavorixafor, a CXCR4 antagonist, can treat certain rare diseases. The CXCR4 protein is a chemokine receptor that is common to both hematopoietic and nonhematopoietic...
by CheckRare Staff | Feb 15, 2022
David Birch, PhD, Scientific Director at the Retina Foundation of the Southwest, discusses phase 2/3 Sirius and Celeste clinical trials of investigational therapy, QR-421a, for people with USH2A-mediated retinitis pigmentosa and Usher syndrome. Retinitis...
by Peter Ciszewski | Feb 15, 2022
Monica Fay, PharmD, Senior Vice President of Global Medical Affairs at Apellis Pharmaceuticals, provides an overview of paroxysmal nocturnal hemoglobinuria (PNH). As Dr. Fay explains, PNH is a rare, life-threatening blood disorder characterized by hemolysis....
by James Radke | Feb 14, 2022
Jonathan Wall, PhD, Director of the University of Tennessee Graduate School of Medicine’s Amyloidosis and Cancer Theranostics Program and Co-founder & Interim Chief Scientific Officer at Attralus, discusses data from a phase 1/2 trial evaluating the ability...
