North Star Ambulatory Assessment changes in ambulant Duchenne boys amenable to skip exons 44, 45, 51, and 53: A 3 year follow up

• Giorgia Coratti ,

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

  ¶‡ These authors are joint first authors on this work.

  Affiliations Pediatric Neurology, Department of Woman and Child Health and Public Health, Child Health Area, Università Cattolica del Sacro Cuore, Rome, Italy, Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

  https://orcid.org/0000-0001-6666-5628

  ⨯
• Marika Pane ,

  Roles Conceptualization, Funding acquisition, Investigation, Methodology, Resources, Writing – original draft, Writing – review & editing

  ¶‡ These authors are joint first authors on this work.

  Affiliations Pediatric Neurology, Department of Woman and Child Health and Public Health, Child Health Area, Università Cattolica del Sacro Cuore, Rome, Italy, Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

  ⨯
• Claudia Brogna ,

  Roles Conceptualization, Data curation, Formal analysis, Methodology, Writing – original draft, Writing – review & editing

  ¶‡ These authors are joint first authors on this work.

  Affiliations Pediatric Neurology, Department of Woman and Child Health and Public Health, Child Health Area, Università Cattolica del Sacro Cuore, Rome, Italy, Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

  ⨯
• Valeria Ricotti,

  Roles Data curation, Methodology, Resources, Writing – original draft, Writing – review & editing

  Affiliations Dubowitz Neuromuscular Centre, UCL & Great Ormond Street Hospital, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

  ⨯
• Sonia Messina,

  Roles Data curation, Methodology, Resources, Writing – original draft, Writing – review & editing

  Affiliations Department of Clinical and Experimental Medicine, University of Messina, Messina, Italy, Nemo SUD Clinical Centre, University Hospital “G. Martino”, Messina, Italy

  ⨯
• Adele D’Amico,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children’s Hospital, Rome, Italy

  ⨯
• Claudio Bruno,

  Roles Data curation, Methodology, Validation, Writing – original draft

  Affiliation Center of Translational and Experimental Myology, IRCCS Istituto Giannina Gaslini, Genoa, Italy

  ⨯
• Gianluca Vita,

  Roles Data curation, Investigation, Methodology, Visualization, Writing – original draft

  Affiliation Nemo SUD Clinical Centre, University Hospital “G. Martino”, Messina, Italy

  https://orcid.org/0000-0003-4190-8042

  ⨯
• Angela Berardinelli,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation IRCCS Mondino Foundation, Pavia, Italy

  ⨯
• Elena Mazzone,

  Roles Data curation, Investigation, Methodology, Resources, Writing – original draft

  Affiliation Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

  ⨯
• Francesca Magri,

  Roles Data curation, Investigation, Visualization, Writing – original draft

  Affiliation Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Dino Ferrari Centre, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

  ⨯
• Federica Ricci,

  Roles Data curation, Methodology, Visualization, Writing – original draft

  Affiliation Neuromuscular Center, AOU Città della Salute e della Scienza, University of Turin, Torino, Italy

  ⨯
• Tiziana Mongini,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Neuromuscular Center, AOU Città della Salute e della Scienza, University of Turin, Torino, Italy

  ⨯
• Roberta Battini,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliations Department of Developmental Neuroscience, Stella Maris Institute, Pisa, Italy, Department of Clinical and Experimental Medicine, University of Pisa, Pisa, Italy

  ⨯
• Luca Bello,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Department of Neurosciences, University of Padua, Padua, Italy

  ⨯
• Elena Pegoraro,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Department of Neurosciences, University of Padua, Padua, Italy

  ⨯
• Giovanni Baranello,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliations Dubowitz Neuromuscular Centre, UCL & Great Ormond Street Hospital, London, United Kingdom, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy

  ⨯
• Stefano C. Previtali,

  Roles Data curation, Investigation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Division of Neuroscience, IRCCS San Raffaele Scientific Institute, Milan, Italy

  https://orcid.org/0000-0003-2546-4357

  ⨯
• Luisa Politano,

  Roles Data curation, Investigation, Writing – original draft, Writing – review & editing

  Affiliation Cardiomyology and Medical Genetics, University of Campania Luigi Vanvitelli, Naples, Italy

  https://orcid.org/0000-0002-0925-7158

  ⨯
• Giacomo P. Comi,

  Roles Data curation, Investigation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Dino Ferrari Centre, Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

  ⨯
• Valeria A. Sansone,

  Roles Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation The NEMO Center in Milan, Neurorehabilitation Unit, ASST Niguarda Hospital, University of Milan, Milan, Italy

  ⨯
• Alice Donati,

  Roles Conceptualization, Formal analysis, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Metabolic Unit, A. Meyer Children’s Hospital, Florence, Italy

  ⨯
• Jean Yves Hogrel,

  Roles Data curation, Investigation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Institute I-Motion, Hôpital Armand Trousseau, Institute of Myology, Paris, France

  ⨯
• Volker Straub,

  Roles Data curation, Investigation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation 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

  ⨯
• Silvana De Lucia,

  Roles Investigation, Methodology, Validation, Visualization

  Affiliation Institute I-Motion, Hôpital Armand Trousseau, Institute of Myology, Paris, France

  ⨯
• Erik Niks,

  Roles Investigation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Department of Neurology, Leiden University Medical Center, Leiden, The Netherlands

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

  ⨯
• Laurent Servais,

  Roles Data curation, Investigation, Methodology, Writing – original draft

  Affiliations Centre de Référence Des Maladies Neuromusculaires, CHU de Liège, Liège, Belgium, Department of Paediatrics, MDUK Neuromuscular Center, University of Oxford, Oxford, United Kingdom

  ⨯
• Imelda De Groot,

  Roles Data curation, Formal analysis, Methodology, Writing – original draft

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

  ⨯
• Mary Chesshyre,

  Roles Data curation, Formal analysis, Methodology, Supervision, Writing – original draft

  Affiliations Dubowitz Neuromuscular Centre, UCL & Great Ormond Street Hospital, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

  ⨯
• Enrico Bertini,

  Roles Conceptualization, Data curation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Disorders, Bambino Gesù Children’s Hospital, Rome, Italy

  ⨯
• Nathalie Goemans,

  Roles Conceptualization, Investigation, Methodology, Writing – original draft, Writing – review & editing

  Affiliation Department of Child Neurology, University Hospitals Leuven, Leuven, Belgium

  https://orcid.org/0000-0002-5157-7155

  ⨯
• Francesco Muntoni,

  Roles Conceptualization, Data curation, Funding acquisition, Methodology, Writing – original draft, Writing – review & editing

  Affiliations Dubowitz Neuromuscular Centre, UCL & Great Ormond Street Hospital, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom

  ⨯
• Eugenio Mercuri ,

  Roles Conceptualization, Data curation, Funding acquisition, Methodology, Supervision, Writing – original draft, Writing – review & editing

  * E-mail: eugeniomaria.mercuri@unicatt.it

  Affiliations Pediatric Neurology, Department of Woman and Child Health and Public Health, Child Health Area, Università Cattolica del Sacro Cuore, Rome, Italy, Centro Clinico Nemo, Fondazione Policlinico Universitario Agostino Gemelli IRCCS, Rome, Italy

  https://orcid.org/0000-0002-9851-5365

  ⨯
•  [ ... ],
• on behalf on the International DMD Group and the iMDEX Consortium

  ¶Membership of the International DMD group and the iMDEX consortium is provided in the Acknowledgments.

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