by James Radke, PhD | May 23, 2022
Olaf Bodamer, MD, PhD, Associate Chief of Genetics and Genomics at Boston Children’s Hospital, discusses how the National Organization for Rare Disorders’ (NORD) Rare Disease Centers of Excellence addresses unmet needs of the rare disease community. There...
by James Radke, PhD | May 4, 2022
Gregory Grabowski, MD, Professor Emeritus at University of Cincinnati College of Medicine highlights the latest research about Gaucher disease presented at WORLDSymposium 2022. This free CME program provides expert analysis on the clinical relevance of this...
by James Radke, PhD | Apr 29, 2022
The U.S. Food and Drug Administration (FDA) has approved mavacamten (Camzyos) for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM). HCM is a genetic cardiovascular disease...
by James Radke, PhD | Apr 26, 2022
Axel Bolte, MSc, MBA, Co-Founder, President, and Chief Executive Officer, Inozyme Pharmaceuticals, gives an overview of ENPP1 deficiency. The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase / phosphodiesterase 1 (ENPP1), which...
by James Radke, PhD | Apr 20, 2022
Bruce Cree, MD, PhD, MAS, Associate Professor of Clinical Neurology in the Department of Neurology at the University of California San Francisco, discusses post-hoc analysis data of newly presenting neuromyelitis optica spectrum disorder (NMOSD) patients from...
