title: Challenges of clinical trial design for DMD.
authors.affiliation: Dubowitz Neuromuscular Centre, UCL Institute of Child Health and Great Ormond Street Hospital, London, UK. Electronic address: v.ricotti@ucl.ac.uk.
authors.forename: Valeria
authors.initials: V
authors.lastname: Ricotti
authors.affiliation: Dubowitz Neuromuscular Centre, UCL Institute of Child Health and Great Ormond Street Hospital, London, UK.
authors.forename: Francesco
authors.initials: F
authors.lastname: Muntoni
authors.affiliation: NIHR Biomedical Research Centre, UCL Institute of Child Health and Great Ormond Street Hospital, London, UK.
authors.forename: Thomas
authors.initials: T
authors.lastname: Voit
mesh_terms: D000818:Animals; D002986:Clinical Trials as Topic; D006801:Humans; D008297:Male; D020388:Muscular Dystrophy, Duchenne / Q000188:drug therapy*; D010641:Phenotype