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

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Use this link to cite this item : https://ir.lib.shimane-u.ac.jp/52903

language	eng
Author	Shiraishi , Hideaki Department of Pediatrics, Hokkaido University School of Medicine, North 15, West 7, Kita-ku, Sapporo 060-8638, Japan YAMADA, Kenji 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 Yamaguchi, Seiji Sato, Norihiro Hokkaido University Hospital Clinical Research and Medical Innovation Center, North 14, West 5, Kita-ku, Sapporo 060-8648, Japan
Subject	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
Journal Title	MOLECULAR GENETICS AND METABOLISM REPORTS
Volume	20
ISSN（Online）	2214-4269
Published Date	2019-09
DOI	10.1016/j.ymgmr.2019.100496
PubMed ID	31372341
Publisher	ELSEVIER
NII Type	Journal Article
OAI-PMH Set	Faculty of Medicine
Remark	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).