Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

• Claudia Brogna ,

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

  ¶‡ 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

  ⨯
• Giorgia Coratti ,

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

  ¶‡ 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

  ⨯
• Marika Pane ,

  Roles Conceptualization, Funding acquisition, Methodology, Supervision, Validation, Writing – original draft

  ¶‡ These authors are Joint First Authors on this work.

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

  ⨯
• Valeria Ricotti,

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

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

  ⨯
• Claudio Bruno,

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

  Affiliation Center of Myology and Neurodegenerative Disorders, Istituto Giannina Gaslini, Genoa, Italy

  ⨯
• Gianluca Vita,

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

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

  ⨯
• Angela Berardinelli,

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

  Affiliation Child Neurology and Psychiatry Unit, ‘‘Casimiro Mondino” Foundation, Pavia, Italy

  ⨯
• Elena Mazzone,

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

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

  ⨯
• Francesca Magri,

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

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

  ⨯
• Federica Ricci,

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

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

  ⨯
• Tiziana Mongini,

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

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

  ⨯
• Roberta Battini,

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

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

  ⨯
• Elena Pegoraro,

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

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

  ⨯
• Giovanni Baranello,

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

  Affiliation Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy

  http://orcid.org/0000-0003-4871-6692

  ⨯
• Stefano C. Previtali,

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

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

  ⨯
• Luisa Politano,

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

  Affiliation Dipartimento di Medicina Sperimentale, Seconda Università di Napoli, Napoli, Italy

  ⨯
• Giacomo P. Comi,

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

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

  ⨯
• Alice Donati,

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

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

  ⨯
• Enrico Bertini,

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

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

  http://orcid.org/0000-0001-9276-4590

  ⨯
• Francesco Muntoni,

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

  Affiliation Dubowitz Neuromuscular Centre, UCL & Great Ormond Street Hospital, London, United Kingdom

  ⨯
• Nathalie Goemans,

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

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

  ⨯
• Eugenio Mercuri ,

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

  * 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

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

  ⨯
•  [ ... ],
• on behalf on the International DMD group

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

  ⨯
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