Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial

• Francesco Muntoni ,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  * E-mail: f.muntoni@ucl.ac.uk

  Affiliation NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, & Great Ormond Street Hospital Trust, London, United Kingdom

  https://orcid.org/0000-0002-9102-5232

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• Michela Guglieri,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation The John Walton Muscular Dystrophy Research Centre, Newcastle University and Newcastle Hospitals NHS Foundation Trust, Newcastle upon Tyne, United Kingdom

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• Jean K. Mah,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Cumming School of Medicine, University of Calgary, Alberta Children’s Hospital, Calgary, Alberta, Canada

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• Kathryn R. Wagner,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Center for Genetic Muscle Disorders, and Departments of Neurology and Neuroscience, Kennedy Krieger Institute, Johns Hopkins School of Medicine, Baltimore, Maryland, United States of America

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• John F. Brandsema,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, United States of America

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• Russell J. Butterfield,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation University of Utah School of Medicine, Salt Lake City, Utah, United States of America

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• Craig M. McDonald,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Lawson Health Research Institute, Children’s Hospital, London, Ontario, Canada

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• Anna G. Mayhew,

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Institute of Genetic Medicine, Newcastle University, Newcastle, United Kingdom

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• Jeffrey P. Palmer,

  Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Software, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Pfizer Inc, Cambridge, Massachusetts, United States of America

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• Shannon Marraffino,

  Roles Conceptualization, Data curation, Funding acquisition, Investigation, Methodology, Project administration, Resources, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Pfizer Inc, Cambridge, Massachusetts, United States of America

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• Lawrence Charnas,

  Roles Conceptualization, Data curation, Funding acquisition, Investigation, Methodology, Project administration, Resources, Software, Supervision, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Pfizer Inc, Cambridge, Massachusetts, United States of America

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• Eugenio Mercuri

  Roles Conceptualization, Investigation, Validation, Visualization, Writing – original draft, Writing – review & editing

  Affiliation Paediatric Neurology, Catholic University, and Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

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