About the Authors
- Irina T. Zaharieva
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Affiliation The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom
- Mattia Calissano
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Affiliation The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom
- Mariacristina Scoto
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Affiliation The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom
- Mark Preston
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Affiliation Department of Pathogen Molecular Biology, London School of Hygiene and Tropical Medicine, London, United Kingdom
- Sebahattin Cirak
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Affiliations The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom, Childrens National Medical Centre, Research Centre for Genetic Medicine, Washington DC, United States of America
- Lucy Feng
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Affiliation The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom
- James Collins
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Affiliation Department of Pediatric Neurology, Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio, United States of America
- Ryszard Kole
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Affiliation Sarepta Therapeutics, Cambridge, Massachusetts, United States of America
- Michela Guglieri
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Affiliation Institute of Human Genetics, Newcastle University, Newcastle, United Kingdom
- Volker Straub
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Affiliation Institute of Human Genetics, Newcastle University, Newcastle, United Kingdom
- Kate Bushby
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Affiliation Institute of Human Genetics, Newcastle University, Newcastle, United Kingdom
- Alessandra Ferlini
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Affiliation Department of Medical Science, University of Ferrara, Ferrara, Italy
- Jennifer E. Morgan
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Affiliation The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom
- Francesco Muntoni
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* E-mail: f.muntoni@ucl.ac.uk
Affiliation The Dubowitz Neuromuscular Centre, Institute of Child Health, University College London, London, United Kingdom
Competing Interests
FM has served on scientific advisory boards for Acceleron Pharma, Genzyme, AVI BioPharma, Debiopharma Group, GlaxoSmithKline, Prosensa, and Santhera Pharmaceutical, has received research support from AVI BioPharma, and has received funding for trials from AVI, Trophos and PTC. RK is employed full time as Distinguished Scientist by Sarepta Therapeutics and owns Sarepta stock. KB has served on scientific advisory boards for Acceleron, AMT, AVI Biopharma, Debiopharm, Genzyme, GlaxoSmithKline, Prosensa, PTC and Santhera and has received funding for trials from AVI and PTC. VS has served on scientific advisory boards for Acceleron, Prosensa, Santhera Pharmaceutical, NicOx, Pfizer and Genzyme and has received funding for trials from GlaxoSmithKline, Prosensa and Genzyme. AF is Principal Investigator of GSK and Prosensa Therapeutics ongoing trials in Italy. This does not alter the authors' adherence to all the PLOS ONE policies on sharing data and materials. All other authors declare that they have no conflicts of interest.
Author Contributions
Conceived and designed the experiments: FM JEM ITZ. Performed the experiments: ITZ MC. Analyzed the data: MP ITZ AF. Contributed reagents/materials/analysis tools: LF. Wrote the paper: ITZ FM JEM. Recruited patients and collected clinical data: MS SC JC. Analysed the results in the intramuscular and systemic clinical trials: RK SC. Recruited patients included in the intramuscular and systemic clinical trials and collected clinical data: SC MG VS KB. Critically reviewed the manuscript: ITZ MC MS MP SC LF JC RK MG VS KB AF JEM FM.