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