Therapy Development for Duchene Muscular Dystrophy: A long and winding road
In the past decades we have seen the rise and fall of a number of promising therapies for Duchenne muscular dystrophy. Despite huge efforts in the field only a few have made it through approval by regulatory agencies. Two main reasons of failure are :1) a limited efficacy of the compounds in development and 2) issues in trial design (the selection of outcome measures and endpoints, selection of patient population). In this seminar we will discuss the issues in clinical development in DMD and explore how we could potentially improve clinical trial design.
Date: 16 June 2021, 14:00
Venue: Online Zoom seminar
Speaker: Prof. Nathalie Goemans (University Hospitals Leuven)
Organising department: Medical Sciences Division
Organiser contact email address: corinne.betts@paediatrics.ox.ac.uk
Host: Prof Laurent Servais (University of Oxford)
Part of: MDUK Oxford Neuromuscular Centre Seminars
Booking required?: Required
Booking url: https://medsci.zoom.us/meeting/register/tJwuf-6hqT0rHtwWLGjUpeLlGDrlAEIFC5xZ
Cost: Free
Audience: Members of the University and OUH
Editors: Nicole Le Grand, Corinne Betts