About the Authors

Akira Ohtake

Roles Conceptualization, Data curation, Formal analysis, Investigation, Methodology, Project administration, Supervision, Writing – review & editing

☯ These three authors contribute equally..

Affiliations Department of Pediatrics, Saitama Medical University Hospital, Moroyama, Japan, Department of Clinical Genomics, Saitama Medical University Hospital, Moroyama, Japan

Yuichi Abe

Roles Conceptualization, Formal analysis, Investigation, Methodology, Writing – review & editing

Current Address: Division of Neurology, National Center for Child Health and Development, Setagaya, Tokyo, Japan.

☯ These three authors contribute equally..

Affiliation Department of Pediatrics, Saitama Medical University Hospital, Moroyama, Japan

Kei Murayama

Roles Conceptualization, Data curation, Investigation, Methodology, Project administration, Supervision, Writing – review & editing

Current Address: Department of Pediatrics, Juntendo University, Bunkyo, Tokyo, Japan.; Diagnostics and Therapeutic of Intractable Diseases, Intractable Disease Research Center, Graduate School of Medicine, Juntendo University, Bunkyo, Tokyo, Japan.

☯ These three authors contribute equally..

Affiliation Department of Metabolism, Chiba Children’s Hospital, Chiba, Japan

Hideaki Shiraishi

Roles Data curation, Investigation, Writing – review & editing

Affiliations Department of Pediatrics, Hokkaido University Hospital, Sapporo, Japan, Department of Pediatrics, Dokkyo Medical University, Mibu, Japan

Hitoshi Osaka

Roles Data curation, Investigation, Writing – review & editing

Affiliation Department of Pediatrics, Jichi Medical University, Shimotsuke, Japan

Satoko Kumada

Roles Data curation, Investigation, Writing – review & editing

Affiliation Department of Neuro Pediatrics, Tokyo Metropolitan Neurological Hospital, Fuchu, Japan

Tetsuya Ito

Roles Data curation, Investigation, Writing – review & editing

Affiliation Department of Pediatrics, Fujita Health University Hospital, Toyoake, Japan

Hidehito Kondo

Roles Data curation, Writing – review & editing

Affiliation Department of Pediatrics, Japanese Red Cross Society Kyoto Daiichi Hospital, Kyoto, Japan

Yusuke Hamada

Roles Data curation, Investigation, Writing – review & editing

Current Address: Department of Pediatrics, Suita Municipal Hospital, Suita, Japan.

Affiliation Department of Pediatrics, Toyonaka Municipal Hospital, Toyonaka, Japan

Sayaka Suzuki-Ajihara

Roles Data curation, Investigation

Affiliations Department of Pediatrics, Saitama Medical University Hospital, Moroyama, Japan, Department of Clinical Genomics, Saitama Medical University Hospital, Moroyama, Japan

Masato Arao

Roles Investigation

Affiliation Department of Pediatrics, Saitama Medical University Hospital, Moroyama, Japan

Yuki Ueda

Roles Data curation, Writing – review & editing

Affiliation Department of Pediatrics, Hokkaido University Hospital, Sapporo, Japan

Kohei Nagai

Roles Data curation, Investigation

Affiliation Department of Pediatrics, Jichi Medical University, Shimotsuke, Japan

Keiko Ichimoto

Roles Data curation, Investigation

Affiliation Department of Metabolism, Chiba Children’s Hospital, Chiba, Japan

Ayako Matsunaga

Roles Data curation, Investigation

Current Address: Department of Pediatrics, St. Marianna University School of Medicine, Kawasaki, Japan.

Affiliation Department of Metabolism, Chiba Children’s Hospital, Chiba, Japan

Hirofumi Kashii

Roles Data curation, Investigation, Writing – review & editing

Affiliation Department of Neuro Pediatrics, Tokyo Metropolitan Neurological Hospital, Fuchu, Japan

Yoko Nakajima

Roles Data curation, Investigation, Writing – review & editing

Affiliation Department of Pediatrics, Fujita Health University Hospital, Toyoake, Japan

Hitoshi Nakagawa

Roles Conceptualization, Formal analysis, Investigation, Methodology, Visualization, Writing – original draft, Writing – review & editing

Affiliation Department of Clinical Development, SBI Pharmaceuticals, Minato, Tokyo, Japan

Shunichi Kurose

Roles Validation, Writing – review & editing

Affiliation Department of Clinical Development, SBI Pharmaceuticals, Minato, Tokyo, Japan

Kaori Fukuda

Roles Formal analysis, Validation, Writing – review & editing

Affiliation Department of Clinical Development, SBI Pharmaceuticals, Minato, Tokyo, Japan

Kiwamu Takahashi

Roles Conceptualization, Methodology, Project administration, Writing – review & editing

Affiliation Department of Research and Development, SBI Pharmaceuticals, Minato, Tokyo, Japan

Mitsugu Yamauchi

Roles Conceptualization, Funding acquisition, Methodology, Project administration, Writing – review & editing

Affiliation Department of Clinical Development, SBI Pharmaceuticals, Minato, Tokyo, Japan

Motowo Nakajima

Roles Conceptualization, Funding acquisition, Investigation, Methodology, Project administration, Supervision, Writing – original draft, Writing – review & editing

motnakaj@sbigroup.co.jp

Affiliation Department of Clinical Development, SBI Pharmaceuticals, Minato, Tokyo, Japan

Competing Interests

We have read the journal’s policy and the authors of this manuscript have the following competing interests: Hitoshi Nakagawa, Shunichi Kurose, Kaori Fukuda, Kiwamu Takahashi, Mitsugu Yamauchi, and Motowo Nakajima are employees of SBI Pharmaceuticals. SBI Pharmaceuticals supported this clinical study by providing research funds and salaries to its own employees only.