New Antisense Therapy in Development for Pompe Disease

touchNEUROLOGY caught up with Tahseen Mozaffar (University California, Irvine, CA, USA) to discuss the results from the PROPEL study (NCT03729362) investigating the use of AT-GAA for the treatment of late-onset Pompe disease. Questions: Could you tell us a little about late onset Pompe disease and the unmet needs in its treatment? (0:15) What is AT-GAA […]

Antonio Federico (EAN Scientific Committee Chair; Professor, Clinical Neurology and Neurometabolic Diseases, University of Siena, Italy) describes the impact increased migration has had on the pathophysiology of neurological conditions and the need to increase awareness of this amongst neurologists. Questions 1. What has the increased migration of European people taught us about the pathophysiology of […]

Antonio Federico (EAN Scientific Committee Chair; Professor, Clinical Neurology and Neurometabolic Diseases, University of Siena, Italy) discusses the unique challenges that rare neurological diseases bring and how the EAN have a Task Force focused on this important area. Questions 1. Could you tell us a little about the Task Force within the EAN that is […]