Yuko Motohashi¹, Hiroyuki Yajima², Akihiko Ishiyama¹, Eri Takeshita¹, Yoko Kobayashi², Hirofumi Komaki¹, Masayuki Sasaki¹ (1.National Center of Neurology and Psychiatry, Department of Child Neurology, Tokyo, Japan, 2.National Center of Neurology and Psychiatry, Department of Rehabilitation, Tokyo, Japan)

Laurent Servais^1,2, Giovanni Baranello³, John W. Day⁴, Nicolas Deconinck⁵, Eugenio Mercuri⁶, Andrea Klein⁷, Basil Darras⁸, Riccardo Masson^1,2, Heidemarie Kletzl⁹, Muna El-Khairi¹⁰, Timothy Seabrook⁹, Marianne Gerber⁹, Ksenija Gorni⁹, Omar Khwaja⁹ (1.Institute of Myology, Paris, France, 2.Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle, Liège, Belgium, 3.Carlo Besta Neurological Research Institute Foundation, Developmental Neurology Unit, Milan, Italy, 4.Department of Neurology, Stanford University, Palo Alto, CA, USA, 5.Queen Fabiola Children’s University Hospital, Université Libre de Bruxelles, Brussels, Belgium; Neuromuscular Reference Center UZ Ghent; Ghent, Belgium, 6.Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy, 7.University Children’s Hospital Basel, Basel, Switzerland; Inselspital, Bern, Switzerland, 8.Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, 9.Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland, 10.Roche Products Ltd, Welwyn Garden City, UK)

Timothy Seabrook¹, Laurent Servais^2,3, Giovanni Baranello⁴, John W. Day⁵, Nicolas Deconinck⁶, Eugenio Mercuri⁷, Andrea Klein⁸, Basil Darras⁹, Riccardo Masson^2,3, Heidemarie Kletzl¹, Muna El-Khairi¹⁰, Marianne Gerber¹, Ksenija Gorni¹, Omar Khwaja¹ (1.Roche Pharmaceutical Research and Early Development, Roche Innovation Center Basel, Basel, Switzerland, 2.Institute of Myology, Paris, France, 3.Reference Center for Neuromuscular Disease, Centre Hospitalier Régional de La Citadelle, Liège, Belgium, 4.Carlo Besta Neurological Research Institute Foundation, Developmental Neurology Unit, Milan, Italy, 5.Department of Neurology, Stanford University, Palo Alto, CA, USA, 6.Queen Fabiola Children’s University Hospital, Université Libre de Bruxelles, Brussels, Belgium; Neuromuscular Reference Center UZ Ghent; Ghent, Belgium, 7.Paediatric Neurology and Nemo Center, Catholic University and Policlinico Gemelli, Rome, Italy, 8.University Children’s Hospital Basel, Basel, Switzerland; Inselspital, Bern, Switzerland, 9.Boston Children’s Hospital, Harvard Medical School, Boston, MA, USA, 10.Roche Products Ltd, Welwyn Garden City, UK)

John W. Day¹, Claudia Chiriboga², Thomas O. Crawford³, Richard S. Finkel⁴, Kayoko Saito⁵, Mari Matsuo⁵, Tamaki Kato⁵, Authorship Truncated⁶, Douglas M. Sproule⁷, Jerry R. Mendell⁸ (1.Stanford University Medical Center, Stanford, CA, USA, 2.Columbia University Medical Center, New York, NY, USA, 3.Johns Hopkins Medicine, Baltimore, MD, USA, 4.Nemours Childrens Hospital, Orlando, FL, USA, 5.Tokyo Womens Medical University, Tokyo, Japan, 6.Complete author list will be included at presentation, 7.AveXis, Inc., Bannockburn, IL, USA, 8.Center for Gene Therapy Nationwide Childrens Hospital, Columbus, OH, USA)

Roxana Dreghici¹, Kathryn R. Wagner², Brenda L. Wong^3,4, Barry J. Byrne⁵, Cuixia Tian⁴, Leslie K. Jacobsen⁶, Giridhar S. Tirucherai⁶, Michael Rabbia⁷, Heidemarie Kletzl¹, Karl Yen¹, Gautam Sajeev⁸, James Signorovitch^8,9, Susan J. Ward⁹, Michelle Krishnan¹ (1.F. Hoffmann-La Roche, Basel, Switzerland, 2.The Johns Hopkins School of Medicine, Baltimore, MD, USA, 3.UMass Memorial Children’s Medical Center, Worcester, MA, US, 4.Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA, 5.University of Florida, Gainesville, FL, USA, 6.Bristol-Myers Squibb, Princeton, NJ, USA, 7.Genentech Inc, South San Francisco, CA, USA, 8.Analysis Group, Inc., Boston, MA, USA, 9.Collaborative Trajectory Analysis Project(cTAP), Cambridge, MA, USA)

Takahiro Yonekawa^1,2, Sara El-Hattab¹, Kevin P. Campbell¹ (1.Howard Hughes Medical Institute, Department of Molecular Physiology and Biophysics, Department of Neurology, University of Iowa Roy J. and Lucille A. Carver College of Medicine, IA, USA, 2.Department of Pediatrics, Mie University Graduate School of Medicine, Mie, Japan)