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13 results on '"Kawakami, Sanae"'

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

Author

Shiraishi, Hideaki, Yamada, Kenji, Oki, Eishin, Ishige, Mika, Fukao, Toshiyuki, Hamada, Yusuke, Sakai, Norio, Ochi, Fumihiro, Watanabe, Asami, Kawakami, Sanae, Kuzume, Kazuyo, Watanabe, Kenji, Sameshima, Koji, Nakamagoe, Kiyotaka, Tamaoka, Akira, Asahina, Naoko, Yokoshiki, Saki, Miyakoshi, Takashi, Oba, Koji, Isoe, Toshiyuki, Hayashi, Hiroshi, Yamaguchi, Seiji, and Sato, Norihiro

Published
2019
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2. Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan

Author

Yamada, Kenji, Shiraishi, Hideaki, Oki, Eishin, Ishige, Mika, Fukao, Toshiyuki, Hamada, Yusuke, Sakai, Norio, Ochi, Fumihiro, Watanabe, Asami, Kawakami, Sanae, Kuzume, Kazuyo, Watanabe, Kenji, Sameshima, Koji, Nakamagoe, Kiyotaka, Tamaoka, Akira, Asahina, Naoko, Yokoshiki, Saki, Miyakoshi, Takashi, Ono, Kota, Oba, Koji, Isoe, Toshiyuki, Hayashi, Hiroshi, Yamaguchi, Seiji, and Sato, Norihiro

Published
2018
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5. Efficacy of bezafibrate for preventing myopathic attacks in patients with very long-chain acyl-CoA dehydrogenase deficiency

Author

Shiraishi, Hideaki, primary, Yamada, Kenji, additional, Egawa, Kiyoshi, additional, Ishige, Mika, additional, Ochi, Fumihiro, additional, Watanabe, Asami, additional, Kawakami, Sanae, additional, Kuzume, Kazuyo, additional, Watanabe, Kenji, additional, Sameshima, Koji, additional, Nakamagoe, Kiyotaka, additional, Tamaoka, Akira, additional, Asahina, Naoko, additional, Yokoshiki, Saki, additional, Kobayashi, Keiko, additional, Miyakoshi, Takashi, additional, Oba, Koji, additional, Isoe, Toshiyuki, additional, Hayashi, Hiroshi, additional, Yamaguchi, Seiji, additional, and Sato, Norihiro, additional

Published
2021
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6. Clinical course in a patient with myopathic VLCAD deficiency during pregnancy with an affected baby

Author

Yamada, Kenji, primary, Matsubara, Keiichi, additional, Matsubara, Yuko, additional, Watanabe, Asami, additional, Kawakami, Sanae, additional, Ochi, Fumihiro, additional, Kuwabara, Kozue, additional, Mushimoto, Yuichi, additional, Kobayashi, Hironori, additional, Hasegawa, Yuki, additional, Fukuda, Seiji, additional, Yamaguchi, Seiji, additional, and Taketani, Takeshi, additional

Published
2019
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7. Open-label clinical trial of bezafibrate treatment in patients with fatty acid oxidation disorders in Japan; 2nd report QOL survey

Author

Shiraishi , Hideaki, YAMADA, Kenji, Oki, Eishin, Ishige, Mika, Fukao, Toshiyuki, Hamada, Yusuke, Sakai, Norio, Ochi Fumihiro, Watanabe, Asami, Kawakami, Sanae, Kuzume, Kazuyo, Watanabe, Kenji, Sameshima, Koji, Nakamagoe, Kiyotaka, Tamaoka, Akira, Asahina, Naoko, Yokoshiki, Saki, Miyakoshi, Takashi, Oba, Koji, Isoe, Toshiyuki, Hayashi, Hiroshi, Yamaguchi, Seiji, Sato, Norihiro, Shiraishi , Hideaki, YAMADA, Kenji, Oki, Eishin, Ishige, Mika, Fukao, Toshiyuki, Hamada, Yusuke, Sakai, Norio, Ochi Fumihiro, Watanabe, Asami, Kawakami, Sanae, Kuzume, Kazuyo, Watanabe, Kenji, Sameshima, Koji, Nakamagoe, Kiyotaka, Tamaoka, Akira, Asahina, Naoko, Yokoshiki, Saki, Miyakoshi, Takashi, Oba, Koji, Isoe, Toshiyuki, Hayashi, Hiroshi, Yamaguchi, Seiji, and Sato, Norihiro

Published
2019

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

Author

1000080374411, Shiraishi, Hideaki, Yamada, Kenji, Oki, Eishin, 1000090420950, Ishige, Mika, 1000070260578, Fukao, Toshiyuki, Hamada, Yusuke, Sakai, Norio, 1000030637905, Ochi, Fumihiro, Watanabe, Asami, Kawakami, Sanae, Kuzume, Kazuyo, Watanabe, Kenji, Sameshima, Koji, 1000010436272, Nakamagoe, Kiyotaka, 1000050192183, Tamaoka, Akira, Asahina, Naoko, Yokoshiki, Saki, Miyakoshi, Takashi, 1000030422926, Oba, Koji, Isoe, Toshiyuki, 1000010622532, Hayashi, Hiroshi, 1000060144044, Yamaguchi, Seiji, Sato, Norihiro, 1000080374411, Shiraishi, Hideaki, Yamada, Kenji, Oki, Eishin, 1000090420950, Ishige, Mika, 1000070260578, Fukao, Toshiyuki, Hamada, Yusuke, Sakai, Norio, 1000030637905, Ochi, Fumihiro, Watanabe, Asami, Kawakami, Sanae, Kuzume, Kazuyo, Watanabe, Kenji, Sameshima, Koji, 1000010436272, Nakamagoe, Kiyotaka, 1000050192183, Tamaoka, Akira, Asahina, Naoko, Yokoshiki, Saki, Miyakoshi, Takashi, 1000030422926, Oba, Koji, Isoe, Toshiyuki, 1000010622532, Hayashi, Hiroshi, 1000060144044, Yamaguchi, Seiji, and Sato, Norihiro

Abstract

Introduction: Fatty acid oxidation disorders (FAODs) are rare diseases caused by a defective mitochondrial fatty acid oxidation (FAO) enzyme. We recently reported that bezafibrate improved patient quality of life (QOL) based on the SF 36 questionnaire score in patients with FAODs during a 50-week, open-label, clinical trial. Herein we conducted further survey assessments of the trial patients to define the long-term efficacy and safety of bezafibrate. Materials and methods: This trial was an open-label, non-randomized, and multicenter study of bezafibrate treatment in five patients with very long-chain acyl-CoA dehydrogenase (VLCAD) deficiency and one patient with carnitine palmitoyltransferase-II (CPT-2) deficiency (median age, 15.9 years; range, 5.8-26.4 years). The bezafibrate administration was continued for a further 102 174 weeks after the 24-week treatment described in our previous study. QOL was quantitated using the 36-Item Short Form Health Survey (SF 36) questionnaire, which constitutes eight components: physical functioning (PF), role limitation due to physical problems, bodily pain, general health perception, vitality, social functioning, role limitation due to emotional problems, and mental health. Results: PF was elevated in all patients and continued to rise during the study, with the total QOL scores increased from baseline in five of the six cases. In particular, three patients older than 20 years showed treatment efficacy, and all subcategories of QOL were elevated in two of these cases. Conclusion: Our findings supported one of the stated benefits of bezafibrate in improving QOL for patients with FAODs.

Published
2019

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