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.
|Download document||Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy|
|Status||draft: consultation open|
|Consultation start date||01/03/2013|
|Consultation end date||31/08/2013|
|Email address for firstname.lastname@example.org|