John W Day, Claudia A Chiriboga, Thomas O Crawford, Basil T Darras, Richard S Finkel, Anne M Connolly, Susan T Iannaccone, Nancy L Kuntz, Loren DM Pena, Meredith Schultz, Perry B Shieh, Edward C Smith, Michelle Farrar*, Douglas E Feltner, Francis G Ogrinc, Thomas A Macek, Elaine Kernbauer, Lynlee M Muehring, James L’Italien, Douglas M Sproule, Brian K Kaspar, Jerry R Mendell. Department of Neurology, Stanford University Medical Center, Stanford, CA, USA; Division of Pediatric Neurology, Columbia University Medical Center, New York, USA; Department of Neurology, Johns Hopkins Medicine, Baltimore, MD, USA; Department of Neurology, Boston Children’s Hospital, Boston, MA, USA; Division of Neurology, Department of Pediatrics, Nemours Children’s Hospital, Orlando, FL, USA; Department of Neurology, Washington University School of Medicine, St. Louis, MO, USA; Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX, USA; Division of Neurology, Ann and Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA; Division of Human Genetics, Cincinnati Children’s Hospital, Cincinnati, OH, USA; AveXis, Inc., Bannockburn, IL, USA; Department of Neurology, David Geffen School of Medicine at UCLA, Los Angeles, CA, USA; Department of Pediatrics, Duke University School of Medicine, Durham, CA, USA; Department of Neurology, Sydney Children’s Hospital, Randwick, NSW, Australia; Center for Gene Therapy Nationwide Children’s Hospital, Columbus, OH, USA; Department of Pediatrics, Ohio State University, Columbus, OH, USA; Department of Neurology, Ohio State University, Columbus, OH, USA