by Peter Ciszewski | Mar 1, 2022
Paula Ragan, PhD, CEO and President, X4 Pharmaceuticals, discusses data presented at The American Society of Hematology Meeting & Exposition (ASH 2021) on a phase 2 open-label extension study of mavorixafor for the treatment of WHIM syndrome. WHIM syndrome...
by CheckRare Staff | Mar 1, 2022
James Howard Jr., MD, Distinguished Professor of Neuromuscular Disease and Professor of Neurology and Medicine at UNC School of Medicine, reviews the treatment landscape for myasthenia gravis. Myasthenia gravis is a chronic autoimmune neuromuscular disease...
by Peter Ciszewski | Mar 1, 2022
Data sharing is a common request in the rare disease community. Unfortunately, the realities of our current research infrastructure make it problematic for researchers to share their data. The net result is a plethora of data silos that can dramatically delay...
by CheckRare Staff | Feb 28, 2022
David Weinstein, MD, Senior Vice President of Clinical Development at Passage Bio, discusses new interim clinical data from the Imagine-1 study of PBGM01, a gene therapy for GM1 gangliosidosis. This data was recently presented at WORLDSymposium 2022. GM1...
by Peter Ciszewski | Feb 28, 2022
David Kronn, MD, Associate Professor of Pathology and Pediatrics at New York Medical College, provides an overview of the CME program that he and Jerry Vockley, MD, PhD, Chief of Genetic and Genomic Medicine at the University of Pittsburgh are hosting that...
