Clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy
Table of contents
This document provides guidance on the development of any medicinal product for the treatment of Duchenne or Becker muscular dystrophy. It addresses the identification of the target population, study design and choice of efficacy endpoints and safety parameters.
Keyword: Duchenne and Becker muscular dystrophy, paediatric population, genetic neuromuscular disorder, molecular diagnosis, motor function, muscle strength
-
List item
Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy (PDF/206.53 KB)
Adopted
First published: 21/12/2015
Last updated: 05/01/2016
EMA/CHMP/236981/2011 Corr.1 -
List item
Overview of comments received on 'Guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy' (PDF/777.55 KB)
First published: 11/03/2016
Last updated: 11/03/2016
EMA/30262/2016 -
List item
Draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy (PDF/200.14 KB)
Draft: consultation closed
First published: 01/03/2013
Last updated: 01/03/2013
Consultation dates: 01/03/2013 to 31/08/2013
EMA/CHMP/236981/2011 -
List item
Concept paper on the need for a guideline on the treatment of Duchenne and Becker muscular dystrophy (PDF/143.74 KB)
Draft: consultation closed
First published: 08/07/2011
Last updated: 08/07/2011
Consultation dates: 23/06/2011 to 30/09/2011
EMA/CHMP/CNSWP/236981/2011