Humans; Treatment Outcome; Muscular Dystrophy, Duchenne/drug therapy; Muscular Dystrophy, Duchenne/genetics; Muscular Dystrophy, Duchenne; Biochemistry, Genetics and Molecular Biology (all); General Biochemistry, Genetics and Molecular Biology; General Medicine
Disciplines :
Neurology
Author, co-author :
Servais, Laurent ; Université de Liège - ULiège ; Department of Paediatrics, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, University of Oxford, Oxford, UK. laurent.servais@paediatrics.ox.ac.uk
Eggenspieler, Damien; SYSNAV, Paris, France
Poleur, Margaux ; Centre Hospitalier Universitaire de Liège - CHU > > Service de neurologie
Grelet, Marc; SYSNAV, Paris, France
Muntoni, Francesco; Dubowitz Neuromuscular Centre, NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, London, UK
Strijbos, Paul; F. Hoffmann-La Roche Ltd, Basel, Switzerland
Annoussamy, Mélanie; SYSNAV, Paris, France
Language :
English
Title :
First regulatory qualification of a digital primary endpoint to measure treatment efficacy in DMD.
The authors thank the collaborators, investigators, patients and family members who have contributed to previous and ongoing SV95C studies. This Correspondence article is dedicated to M. Annoussamy, who could not approve the final version, as she sadly passed away shortly before the final draft; the authors are immensely grateful for her leadership and invaluable efforts during the EMA qualification process. Medical writing support was provided by J. Ciarochi of Nucleus Global, in accordance with Good Publication Practice (GPP) 2022 guidelines ( https://www.ismpp.org/gpp-2022 ) and was funded by F. Hoffmann-La Roche Ltd.
