Naarding, Karin;
Stimpson, Georgia;
Ward, Susan J;
Goemans, Nathalie;
McDonald, Craig;
Mercuri, Eugenio;
Muntoni, Francesco;
(2023)
269th ENMC International Workshop:10 years of Clinical trials in DMD – What have we learned? 9–11 December 2022, The Netherlands.
Neuromuscular Disorders
, 33
(11)
pp. 897-910.
10.1016/j.nmd.2023.10.003.
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Abstract
There are multiple avenues for therapeutic development in Duchenne Muscular Dystrophy (DMD), which are highlighted in the first section of this report for the “10 years of Clinical trials in DMD – What have we learned?” workshop. This report then provides an overview of the presentations made at the workshop grouped into the following core themes: trial outcomes, disease heterogeneity, meaningfulness of outcomes and the utility of real-world data in trials. Finally, we present the consensus that was achieved at the workshop on the learning points from 10 years of clinical trials in DMD, and possible action points from these. This includes further work in expanding the scope and range of trial outcomes and assessing the efficacy of new trial structures for DMD. We also highlight several points which should be addressed during future interactions with regulators, such as clinical meaningfulness and the use of real-world data.
| Type: | Article |
|---|---|
| Title: | 269th ENMC International Workshop:10 years of Clinical trials in DMD – What have we learned? 9–11 December 2022, The Netherlands |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1016/j.nmd.2023.10.003 |
| Publisher version: | https://doi.org/10.1016/j.nmd.2023.10.003 |
| Language: | English |
| Additional information: | This version is the author accepted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. |
| Keywords: | Duchenne muscular dystrophy, clinical trial, outcome measure, real world data, meaningfulness |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10179891 |
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