Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan; 2nd report QOL survey

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言語	英語
著者	Shiraishi , Hideaki Department of Pediatrics, Hokkaido University School of Medicine, North 15, West 7, Kita-ku, Sapporo 060-8638, Japan 山田健治 Oki, Eishin Department of Pediatrics, Tsugaru General Hospital, 12-3, Iwaki-cho, Goshogawara, Aomori 037-0074, Japan Ishige, Mika Department of Pediatrics and Child Health, Nihon University School of Medicine, 1-6, Kanda-Surugadai, Chiyoda-ku, Tokyo 101-8309, Japan Fukao, Toshiyuki Department of Pediatrics, Graduate School of Medicine, Gifu University, 1-1, Yanagido, Gifu 501-1194, Japan Hamada, Yusuke Child Healthcare and Genetic Science Laboratory, Division of Health Sciences, Osaka University Graduate School of Medicine, 1-7 Yamada-oka, Suita, Osaka 565-0871, Japan　/　Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, 4-2-78, Fukushima, Fukushima-ku, Osaka 553-0003, Japan Sakai, Norio Child Healthcare and Genetic Science Laboratory, Division of Health Sciences, Osaka University Graduate School of Medicine, 1-7 Yamada-oka, Suita, Osaka 565-0871, Japan　/　Department of Pediatrics, Osaka Hospital, Japan Community Healthcare Organization, 4-2-78, Fukushima, Fukushima-ku, Osaka 553-0003, Japan Ochi Fumihiro Department of Pediatrics, Yawatahama City General Hospital, 638, Ohira-ichibankochi, Yawatahama, Ehime 796-8502, Japan　/　Department of Pediatrics, Ehime University Graduate School of Medicine, Shitsukawa, Toon, Ehime 791-0295, Japan Watanabe, Asami Department of Pediatrics, Yawatahama City General Hospital, 638, Ohira-ichibankochi, Yawatahama, Ehime 796-8502, Japan　/　Department of Pediatrics, Ehime University Graduate School of Medicine, Shitsukawa, Toon, Ehime 791-0295, Japan Kawakami, Sanae Department of Pediatrics, Yawatahama City General Hospital, 638, Ohira-ichibankochi, Yawatahama, Ehime 796-8502, Japan Kuzume, Kazuyo Department of Pediatrics, Yawatahama City General Hospital, 638, Ohira-ichibankochi, Yawatahama, Ehime 796-8502, Japan　/　Department of Community and Emergency Medicine, Ehime University School of Medicine, Shitsukawa, Toon, Ehime 791-0295, Japan Watanabe, Kenji Department of Pediatrics, Kagoshima City Hospital, 37-1, Uearata-cho, Kagoshima 890-8760, Japan Sameshima, Koji Department of Pediatrics, Kagoshima City Hospital, 37-1, Uearata-cho, Kagoshima 890-8760, Japan Nakamagoe, Kiyotaka Department of Neurology, Division of Clinical Medicine, Faculty of Medicine, University of Tsukuba, 1-1-1, Tennoudai, Tsukuba, Ibaraki 305-8575, Japan Tamaoka, Akira Department of Neurology, Division of Clinical Medicine, Faculty of Medicine, University of Tsukuba, 1-1-1, Tennoudai, Tsukuba, Ibaraki 305-8575, Japan Asahina, Naoko Department of Pediatrics, Hokkaido University School of Medicine, North 15, West 7, Kita-ku, Sapporo 060-8638, Japan Yokoshiki, Saki Hokkaido University Hospital Clinical Research and Medical Innovation Center, Research and Development Division, North 14, West 5, Kita-ku, Sapporo 060-8648, Japan Miyakoshi, Takashi Hokkaido University Hospital Clinical Research and Medical Innovation Center, Research and Development Division, North 14, West 5, Kita-ku, Sapporo 060-8648, Japan Oba, Koji Department of Biostatistics, School of Public Health, Graduate School of Medicine, The University of Tokyo, 7-3-1, Hongo, Bunkyo-ku, Tokyo 113-0033, Japan Isoe, Toshiyuki Hayashi, Hiroshi Hokkaido University Hospital Clinical Research and Medical Innovation Center, Research and Development Division, North 14, West 5, Kita-ku, Sapporo 060-8648, Japan 山口清次 Sato, Norihiro Hokkaido University Hospital Clinical Research and Medical Innovation Center, North 14, West 5, Kita-ku, Sapporo 060-8648, Japan
主題	Bezafibrate; Fatty acid oxidation disorders (FAODs); Very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency; Carnitine palmitoyltransferase-II (CPT-2) deficiency; Clinical trial; Quality of life
掲載誌名	MOLECULAR GENETICS AND METABOLISM REPORTS
巻	20
ISSN（Online）	2214-4269
発行日	2019-09
DOI	10.1016/j.ymgmr.2019.100496
PubMed ID	31372341
出版者	ELSEVIER
資料タイプ	学術雑誌論文
部局	医学部
備考	This study was supported by The multicenter clinical trial of the efficacy and safety of bezafibrate for mitochondrial fatty acid beta-oxidation disorders (Grant Number, 16lk0103005h0005) from the Japan Agency for Medical Research and Development (AMED).