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...
by James Radke, PhD | Apr 13, 2022
Damian M. May, PharmD, MBA, Senior Director Of Health Economics and Outcomes Research at Acadia Pharmaceuticals, discusses the multidisciplinary approach to care needed for Rett syndrome. Rett syndrome is a rare progressive neurodevelopmental condition that...
by James Radke, PhD | Apr 6, 2022
The U.S. Food and Drug Administration (FDA) has given accelerated approval to Vijoice (alpelisib) for the treatment of patients 2 years of age and older with severe manifestations of PIK3CA-Related Overgrowth Spectrum (PROS) who require systemic therapy. Continued...
