^○Nicola Specchio¹, Angela Schulz², Paul Gissen³, De Los Emily Reyes⁴, Ruth Williams⁵, Heather Cahan⁶, Peter Slasor⁶, David Jacoby⁶ (Bambino Gesu Childrens Hospital, IRCCS, Rome, Italy¹, University Medical Center Hamburg—Eppendorf, Hamburg, Germany², Great Ormond Street Hospital for Children, London, United Kingdom³, Nationwide Children’s Hospital, The Ohio State University, Columbus, OH, United States⁴, Guys and St Thomas NHS Foundation Trust, London, United Kingdom⁵, BioMarin Pharmaceutical Inc., Novato, CA, United States⁶)

^○Yoshikatsu Eto¹, Takashi Miyajima², Junko Igarashi², Keiko Akiyama¹, Hiroko Yanagisawa¹, Hossain Arif¹, Kaoru Eto³, Takeo Iwamoto⁴ (Advanced Clinical Research Center, Institute of Neurological Disorders, Kanagawa, Japan¹, AngesMG Institute for Rare Diseases², Tokyo Women’s Medical University, Department of Pediatrics, Tokyo, Japan³, Tokyo Jikei University School of Medicine, Core Central Laboratory, Tokyo, Japan⁴)

^○Makito Hirano^{1, 2}, Kazumasa Saigoh², Yusaku Nakamura¹, Susumu Kusunoki² (Department of Neurology, Sakai Hospital Kindai University Faculty of Medicine, Osaka, Japan¹, Department of Neurology, Kindai University Facutly of Medicine, Osaka, Japan²)

^○Karin Kojima¹, Akihiko Miyauchi¹, Takeshi Nakajima², Sayaka Asari³, Hiroaki Mizukami⁴, Hitoshi Osaka¹, Shin—ichi Muramatsu^{5, 6, 7}, Takanori Yamagata¹ (The Department of Pediatrics, Jichi Medical University, Tochigi, Japan¹, Department of Neurosurgery, Jichi Medical University, Shimotsuke—shi, Tochigi, Japan², Department of Neurology, Saitama Medical Center, Jichi Medical University, Saitama, Japan³, Division of Genetic Therapeutics, Jichi Medical University, Shimotsuke, Tochigi, Japan⁴, Division of Neurology, Jichi Medical University, Shimotsuke, Tochigi, Japan⁵, Center for Gene & Cell Therapy, The Institute of Medical Science, The University of Tokyo, Japan⁶, Gene Therapy Research Institution Co., Ltd.⁷)

^○Ling Yan¹, Nancy Kuntz², Wildon Farwell³, Zhenshao John Zhong³, Peng Sun³, Sarah Gheuens³, Eugene Schneider⁴, Richard Finkel^5,6 (Clinical Development Head, Biogen Japan¹, Division of Neurology, Ann & Robert H. Lurie Children’s Hospital of Chicago, Chicago, IL, USA², Biogen, Cambridge, MA, USA³, Ionis Pharmaceuticals Inc., Carlsbad, CA, USA⁴, Division of Neurology, Department of Pediatrics, Nemours Children’s Hospital, Orlando, FL, USA^{5, the ENDEAR study group6})

^○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)