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

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

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