Online Seminar: Therapy Development for Duchene Muscular Dystrophy: A long and winding road
Prof. Nathalie Goemans, Dept. of Pediatric Neurology, University Hospitals Leuven, KUL University of Leuven, Belgium
Wednesday, 16 June 2021, 2pm to 3pm
Hosted by Professor Laurent Servais
MDUK OXFORD NEUROMUSCULAR CENTRE SEMINAR SERIES
This seminar is intended for the personnel and students at the University of Oxford and the Oxford University Hospitals Foundation Trust.
Online Seminar – please register using the following link https://medsci.zoom.us/meeting/register/tJwuf-6hqT0rHtwWLGjUpeLlGDrlAEIFC5xZ
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.
Nathalie Goemans is a pediatrician and child neurologist currently emeritus assistant professor at the Faculty of Medicine, University of Leuven, Belgium. As former head of the Neuromuscular Reference Centre for Children at the University Hospitals Leuven, she has devoted her career to the diagnosis and multidisciplinary management of patients with neuromuscular disorders, and to translational and clinical research in therapy development for Duchenne muscular dystrophy and spinal muscular atrophy.