by Peter Ciszewski | Feb 17, 2022
Caroline Hastings, MD, Hematologist-Oncologist and Neuro-Oncologist from the UCSF Benioff Children’s Hospital, explains why finding a treatment for Niemann-Pick Disease Type C (NPC) has been difficult. NPC is a disabling neurogenetic disorder that has...
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 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 Peter Ciszewski | Feb 11, 2022
Dawn Laney, MS, CGC, CCRC, Assistant Professor, and Director of Emory Genetic Clinical Trials Center, describes the MOPPet study, a prospective, multicenter pilot study of Fabry disease clinical and biochemical findings in young pediatric patients. Data from...
by Peter Ciszewski | Feb 9, 2022
Steven Pipe, MD, Professor of Pediatrics and Pathology, and Pediatric Medical Director of the Hemophilia and Coagulation Disorders Program at the University of Michigan, discusses the recent announcement of positive long-term results from the phase 3 HOPE-B...
