Safety, Tolerability, and Pharmacokinetics of SMT C1100, a 2-Arylbenzoxazole Utrophin Modulator, following Single- and Multiple-Dose Administration to Pediatric Patients with Duchenne Muscular Dystrophy

• Valeria Ricotti,

  Affiliation Dubowitz Neuromuscular Centre, UCL Institute of Child Health, London, United Kingdom

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• Stefan Spinty,

  Affiliation Alder Hey Children’s NHS Foundation Trust, Liverpool, United Kingdom

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• Helen Roper,

  Affiliation Birmingham Heartlands Hospital, Heart of England NHS Foundation Trust, Birmingham, United Kingdom

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• Imelda Hughes,

  Affiliation Royal Manchester Children’s Hospital, Central Manchester University Hospitals NHS Foundation Trust, United Kingdom

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• Bina Tejura,

  Affiliation Summit Therapeutics, Abingdon, United Kingdom

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• Neil Robinson,

  Affiliation S.H.B. Enterprises Limited, Beaconsfield, United Kingdom

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• Gary Layton,

  Affiliation ParamStat Limited, Ash, United Kingdom

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• Kay Davies,

  Affiliation MRC Functional Genomics Unit, Department of Physiology Anatomy and Genetics, University of Oxford, United Kingdom

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• Francesco Muntoni ,

  Contributed equally to this work with: Francesco Muntoni, Jonathon Tinsley

  Affiliation Dubowitz Neuromuscular Centre, UCL Institute of Child Health, London, United Kingdom

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• Jonathon Tinsley

  Contributed equally to this work with: Francesco Muntoni, Jonathon Tinsley

  * E-mail: jon.tinsley@summitplc.com

  Affiliation Summit Therapeutics, Abingdon, United Kingdom

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