About the Authors
- Narinder Janghra
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Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guilford Street, London, WC1N1EH, United Kingdom
- Jennifer E. Morgan
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* E-mail: jennifer.morgan@ucl.ac.uk
Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guilford Street, London, WC1N1EH, United Kingdom
- Caroline A. Sewry
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Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guilford Street, London, WC1N1EH, United Kingdom
- Francis X. Wilson
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Affiliation Summit Therapeutics plc, 85b Park Drive, Milton Park, Abingdon, Oxfordshire, OX14 4RY, United Kingdom
- Kay E. Davies
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Affiliation Medical Research Council Functional Genomics Unit, Department of Physiology, Anatomy and Genetics, University of Oxford, Oxford, OX1 3PT, United Kingdom
- Francesco Muntoni
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Affiliation Dubowitz Neuromuscular Centre, Molecular Neurosciences Section, Developmental Neurosciences Programme, UCL Institute of Child Health, 30 Guilford Street, London, WC1N1EH, United Kingdom
- Jonathon Tinsley
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Affiliation Summit Therapeutics plc, 85b Park Drive, Milton Park, Abingdon, Oxfordshire, OX14 4RY, United Kingdom
Competing Interests
This work was funded by Summit Therapeutics and Joining Jack (grant awarded to FM and JEM). JEM is supported by Great Ormond Street Hospital Children’s Charity and FM is funded from the GOSH Biomedical Research Centre. This work was supported by the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children NHS Foundation Trust and University College London. We gratefully acknowledge the support of the Medical Research Council and of the Biomedical Research Centre at GOSH for their support of the Centre for Neuromuscular Diseases Biobank. JT and FXW are employees of the sponsor (Summit Therapeutics). They were involved in the study design, data analysis, decision to publish, reviewing of the manuscript. Francesco Muntoni has served on scientific advisory boards for Roche; PTC; 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 Française Contre les Myopathies (AFM), the Muscular Dystrophy Campaign, the Great Ormond Street Hospital (GOSH) Biomedical Research Centre; Genethon; NIH. His institutions (UCL and GOSH) receive funding for clinical trials from Prosensa/ Biomarin; the British Heart Foundation; Summit Therapeutics, Roche; ISIS; Pfizer and Sarepta and the European Community. Jonathon Tinsley and Francis Wilson are employees of Summit Therapeutics. Kay Davies is a shareholder in Summit Therapeutics. Caroline Sewry serves on the advisor board of Audentes; she is associated editor of Neuropathology Applied Neurobiology and serves on the editorial board of Neuromuscular Disorders and Muscle and Nerve. This does not alter the authors’ adherence to PLOS ONE policies on sharing data and materials.
Author Contributions
Conceived and designed the experiments: NJ JEM CAS FXW KED FM JT. Performed the experiments: NJ. Analyzed the data: NJ JEM CAS FXW KED FM JT. Wrote the paper: NJ JEM CAS FXW KED FM JT.