Clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy
| Current effective version |
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| Reference number | EMA/CHMP/236981/2011, Corr. 11 |
| Published | 21/12/2015 |
| Effective from | 01/07/2016 |
| Keywords | Duchenne and Becker muscular dystrophy, paediatric population, genetic neuromuscular disorder, molecular diagnosis, motor function, muscle strength |
| Description | 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. |
Document history
First version Current version | In operation: 01/07/2016–present Published: 11/03/2016 Published: 01/03/2013 Published: 08/07/2011 |