Clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy

Current effective version

PDF iconAdopted guideline

Reference numberEMA/CHMP/236981/2011, Corr. 11
Effective from01/07/2016
KeywordsDuchenne and Becker muscular dystrophy, paediatric population, genetic neuromuscular disorder, molecular diagnosis, motor function, muscle strength
DescriptionThis 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.

Document history

First version

Current version

PDF iconAdopted guideline

PDF iconOverview of comments

PDF iconDraft guideline

PDF iconConcept paper

In operation: 01/07/2016–present

Published: 11/03/2016

Published: 01/03/2013

Published: 08/07/2011

Related content

How useful was this page?

Add your rating