About the Authors
- Valentina Sardone
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Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Validation, Writing – original draft
Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom
- Matthew Ellis
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Roles Data curation, Formal analysis, Investigation, Methodology, Software, Validation, Writing – review & editing
Affiliation Department of Neurodegenerative Diseases, UCL Institute of Neurology, London, United Kingdom
- Silvia Torelli
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Roles Conceptualization, Formal analysis, Validation, Writing – original draft
Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom
- Lucy Feng
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Roles Investigation, Methodology, Writing – review & editing
Affiliations Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom, Division of Neuropathology, UCL Institute of Neurology, National Hospital for Neurology and Neurosurgery, UCLH NHS Foundation Trust, London, United Kingdom
- Darren Chambers
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Roles Investigation, Methodology, Writing – review & editing
Affiliations Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom, Division of Neuropathology, UCL Institute of Neurology, National Hospital for Neurology and Neurosurgery, UCLH NHS Foundation Trust, London, United Kingdom
- Deborah Eastwood
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Roles Methodology, Writing – review & editing
Affiliations Department of Orthopaedics, Great Ormond Street Hospital, London, United Kingdom, The Royal National Orthopaedic Hospital, Stanmore, United Kingdom
- Caroline Sewry
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Roles Conceptualization, Investigation, Methodology, Supervision, Writing – review & editing
Affiliations Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom, Wolfson Centre for Inherited Neuromuscular Diseases, RJAH Orthopaedic Hospital, Oswestry, United Kingdom
- Rahul Phadke
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Roles Conceptualization, Investigation, Methodology, Supervision, Writing – review & editing
Affiliations Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom, Division of Neuropathology, UCL Institute of Neurology, National Hospital for Neurology and Neurosurgery, UCLH NHS Foundation Trust, London, United Kingdom
- Jennifer E. Morgan
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Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Investigation, Methodology, Supervision, Validation, Writing – original draft
Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom
- Francesco Muntoni
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Roles Conceptualization, Data curation, Formal analysis, Funding acquisition, Investigation, Methodology, Supervision, Validation, Writing – original draft
* E-mail: f.muntoni@ucl.ac.uk
Affiliations Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neuroscience Programme, UCL Great Ormond Street Institute of Child Health, London, United Kingdom, NIHR Great Ormond Street Hospital Biomedical Research Centre, London, United Kingdom
Competing Interests
VS was partially sponsored by Sarepta Therapeutics. FM has served on scientific advisory boards for Roche; Summit Therapeutics; Italfarmaco; Pfizer and Sarepta Therapeutics. He serves on the editorial board of Neuromuscular Disorders and Neuropediatrics. His institution (UCL) receives research support from the European Union, the Medical Research Council, the Wellcome Trust, the Association Francaise Contre les Myopathies (AFM), the Muscular Dystrophy Campaign, the Great Ormond Street Hospital (GOSH) Biomedical Research Centre, Genethon and National Institutes of Health (NIH). His institutions (UCL and GOSH) receive funding for clinical trials from Prosensa/Biomarin, the British Heat Foundation, Summit Therapeutics, Roche, ISIS, Pfizer, Sarepta and the European Community. CAS serves on the advisor board of Audentes. She serves on the editorial board of Neuromuscular Disorders and Neuropathology Applied Neurobiology. This does not alter the authors’ adherence to PLOS ONE policies on sharing data and materials.