^○Specchio Nicola¹, Angela Schulz², Paul Gissen³, De Los Emily Reyes⁴, Ruth Williams⁵, Heather Cahan⁶, Peter Slasor⁶, David Jacoby⁶ (1.Bambino Gesu Childrens Hospital, IRCCS, Rome, Italy, 2.University Medical Center Hamburg-Eppendorf, Hamburg, Germany, 3.Great Ormond Street Hospital for Children, London, United Kingdom, 4.Nationwide Children s Hospital, The Ohio State University, Columbus, OH, United States, 5.Guys and St Thomas NHS Foundation Trust, London, United Kingdom, 6.BioMarin Pharmaceutical Inc., Novato, CA, United States)

^○Yoshikatsu Eto¹, Takashi Miyajima², Junko Igarashi², Keiko Akiyama¹, Hiroko Yanagisawa¹, Arif Hossain¹, Kaoru Eto³, Takeo Iwamoto⁴ (1.Advanced Clinical Research Center, Institute of Neurological Disorders, Kanagawa, Japan, 2.AngesMG Institute for Rare Diseases, 3.Tokyo Women's Medical University, Department of Pediatrics, Tokyo, Japan, 4.Tokyo Jikei University School of Medicine, Core Central Laboratory, Tokyo, Japan)

^○Makito Hirano^1,2, Kazumasa Saigoh², Yusaku Nakamura¹, Susumu Kusunoki² (1.Department of Neurology, Sakai Hospital Kindai University Faculty of Medicine,Osaka,Japan, 2.Department of Neurology, Kindai University Facutly of Medicine, Osaka, Japan)

^○Karin Kojima¹, Akihiko Miyauchi¹, Takeshi Nakajima², Sayaka Asari³, Hiroaki Mizukami⁴, Hitoshi Osaka¹, Shin-ichi Muramatsu⁵, Takanori Yamagata¹ (1.The Department of Pediatrics, Jichi Medical University, Tochigi, Japan, 2.Department of Neurosurgery, Jichi Medical University, Shimotsuke-shi, Tochigi, Japan, 3.Department of Neurology, Saitama Medical Center, Jichi Medical University, Saitama, Japan, 4.Division of Genetic Therapeutics, Jichi Medical University, Shimotsuke, Tochigi, Japan, 5.Department of Neurology, Jichi Medical University, Shimotsuke, Tochigi, Japan, 6.Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo, Japan)

^○Saito Kayoko¹, Nancy Kuntz², Wildon Farwell³, Zhenshao John Zhong³, Peng Sun³, Sarah Gheuens³, Eugene Schneider⁴, Richard Finkel（endear Study Group）⁵ (1.Tokyo Women’s Medical University, Institute of Medical Genetics,Tokyo, Japan, 2.Division of Neurology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA, 3.Biogen, Cambridge, MA, USA, 4.Ionis Pharmaceuticals Inc., Carlsbad, CA, USA, 5.Division of Neurology, Department of Pediatrics, Nemours Children’s Hospital, Orlando, FL, USA)

^○Shohei Kusabiraki, Akihiko Ishiyama, Eri Takeshita, Yuko Shimizu-motohashi, Takashi Saito, Hirofumi Komaki, Eiji Nakagawa, Kenji Sugai, Masayuki Sasaki (Department of Child Neurology, National Center of Neurology and Psychiatry, Tokyo, Japan)