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

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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 closed
First published 2013-03-01
Last updated 2013-03-01

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.