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...
by James Radke, PhD | Mar 31, 2022
Terence R. Flotte, MD, Provost and Executive Deputy Chancellor of the University of Massachusetts Medical School, discusses a phase 1 clinical trial evaluating an AAV gene therapy in patients with Tay-Sachs disease. Tay-Sachs disease is a rare...
by James Radke, PhD | Mar 29, 2022
The U.S. Food and Drug Administration (FDA) has approved fenfluramine (Fintepla) for the treatment of seizures associated with Lennox-Gastaut syndrome in patients 2 years of age and older. Last year, the orphan drug was approved for treating seizures in children with...
by James Radke, PhD | Mar 28, 2022
Dawn Laney, MS, CGC, CCRC, Co-Founder and Chief Genetic Officer of ThinkGenetic, discusses how the company is helping physicians diagnose rare diseases more efficiently. As Ms. Laney explains, ThinkGenetic utilizes firsthand knowledge as genetic counselors as...
