Draft guideline on the clinical investigation of medicinal products for the treatment of Duchenne and Becker muscular dystrophy

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Document details

Download document Draft 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 cnswpsecretariat@ema.europa.eu

Summary

Recent advances in basic and clinical research have opened new perspectives for future therapeutic options in Duchenne and Becker muscular dystrophy. This guideline is intended to provide guidance for the evaluation of medicinal products in the treatment of these diseases, including study design, the choice of appropriate efficacy endpoints and the definition of reliable surrogate outcome measures.