269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands

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.