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 CheckRare Staff | Feb 9, 2022
James Howard Jr, MD, Distinguished Professor of Neuromuscular Disease and Professor of Neurology and Medicine at UNC School of Medicine, discusses the phase 3 trial of efgartigimod in patients with myasthenia gravis, which recently led to the drug’s approval by...
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...
by James Radke, PhD | Feb 8, 2022
Jeffrey Neul, MD, PhD, Professor of Pediatrics at Vanderbilt University Medical Center, discusses recent top-line results from a phase 3 trial testing trofinetide to treat children with Rett syndrome. Trofinetide is an analog of insulin-like growth factor 1...
by Peter Ciszewski | Feb 7, 2022
Phillip Moreau, MD, PhD, Head of the Hematology Department at the University Hospital Hôtel-Dieu, discusses the updated results from MajesTEC-1, a phase 1/2 study of teclistamab in relapsed/refractory multiple myeloma. These results were recently presented at...
