Determining minimal clinically important differences in the North Star Ambulatory Assessment (NSAA) for patients with Duchenne muscular dystrophy

• Vandana Ayyar Gupta ,

  Contributed equally to this work with: Vandana Ayyar Gupta, Jacqueline M. Pitchforth

  Roles Conceptualization, Formal analysis, Investigation, Methodology, Project administration, Visualization, Writing – original draft

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  https://orcid.org/0000-0003-3022-4307

  ⨯
• Jacqueline M. Pitchforth ,

  Contributed equally to this work with: Vandana Ayyar Gupta, Jacqueline M. Pitchforth

  Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Writing – original draft

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Joana Domingos †,

  † Deceased.

  Roles Conceptualization, Methodology

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Deborah Ridout,

  Roles Data curation, Formal analysis, Methodology, Visualization, Writing – review & editing

  Affiliations Population, Policy & Practice Research and Teaching Department, UCL Great Ormond Street (GOS) Institute of Child Health, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

  ⨯
• Mario Iodice,

  Roles Investigation, Project administration, Writing – review & editing

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Catherine Rye,

  Roles Investigation, Project administration, Writing – review & editing

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Mary Chesshyre,

  Roles Methodology, Project administration, Writing – review & editing

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Amy Wolfe,

  Roles Investigation, Project administration, Writing – review & editing

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Victoria Selby,

  Roles Methodology, Writing – review & editing

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Anna Mayhew,

  Roles Conceptualization, Methodology, Supervision, Writing – review & editing

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

  ⨯
• Elena S. Mazzone,

  Roles Conceptualization, Methodology, Writing – review & editing

  Affiliation Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Universita Cattolica del Sacro Cuore, Rome, Italy

  ⨯
• Valeria Ricotti,

  Roles Conceptualization, Writing – review & editing

  Affiliations UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

  ⨯
• Jean-Yves Hogrel,

  Roles Methodology, Writing – review & editing

  Affiliation Institute of Myology, Paris, France

  ⨯
• Erik H. Niks,

  Roles Writing – review & editing

  Affiliations Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands, European Reference Network for Rare Neuromuscular Diseases – ERN EURO NMD

  https://orcid.org/0000-0001-5892-5143

  ⨯
• Imelda de Groot,

  Roles Writing – review & editing

  Affiliation Department of Rehabilitation, Donders Center for Medical Neuroscience, Radboud University Medical Center, Nijmegen, The Netherlands

  ⨯
• Laurent Servais,

  Roles Supervision, Writing – review & editing

  Affiliations Institute of Myology, Paris, France, Division of Child Neurology, Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de Références des Maladies Neuromusculaires, Department of Paediatrics, University Hospital Liège & University of La Citadelle, Liège, Belgium, Department of Paediatrics, MDUK Neuromuscular Center, University of Oxford, Oxford, United Kingdom

  ⨯
• Volker Straub,

  Roles Writing – review & editing

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

  https://orcid.org/0000-0001-9046-3540

  ⨯
• Eugenio Mercuri,

  Roles Conceptualization, Writing – review & editing

  Affiliations Child Neurology Unite Centro Nemo, IRCCS Fondazione Policlinico Gemelli, Universita Cattolica del Sacro Cuore, Rome, Italy, Child Neurology Unit, Universita Cattolica del Sacro Cuore, Rome, Italy

  ⨯
• Adnan Y. Manzur,

  Roles Conceptualization, Writing – review & editing

  Affiliation UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom

  ⨯
• Francesco Muntoni ,

  Roles Conceptualization, Funding acquisition, Methodology, Project administration, Supervision, Writing – review & editing

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

  Affiliations UCL Great Ormond Street Institute of Child Health, Dubowitz Neuromuscular Centre, Great Ormond Street Hospital for Children, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

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•  [ ... ],
• on behalf of the iMDEX Consortium and the U.K. NorthStar Clinical Network

  ¶Membership of the iMDEX Consortium and the U.K. NorthStar Clinical Network is provided in the Acknowledgments.

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