Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy (DBMD). The increasing number of clinical trials that recruit a rather small number of patients for these progressive disorders has raised several issues, including the study design, the choice of appropriate efficacy endpoints in general and the definition of reliable surrogate outcome measures 1, 2 as well as the need of subgroup analyses with respect to the heterogeneous patient population and the duration of the trials.


Document details

Download document Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy
Reference number EMA/CHMP/236981/2011
Status draft: consultation open
First published 01/03/2013
Last updated 01/03/2013
Consultation start date 01/03/2013
Consultation end date 31/08/2013
Email address for submissions


Posted on the EMA website on 1 March 2013