タファミジス：遺伝性トランスサイレチンアミロイドーシスに対する蛋白質安定化剤
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| Published in | Shinzo Vol. 49; no. 5; pp. 438 - 443 |
|---|---|
| Main Authors | , |
| Format | Journal Article |
| Language | Japanese |
| Published |
公益財団法人 日本心臓財団
15.05.2017
Japan Heart Foundation |
| Online Access | Get full text |
| ISSN | 0586-4488 2186-3016 |
| DOI | 10.11281/shinzo.49.438 |
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| Author | 山下, 太郎 安東, 由喜雄 |
|---|---|
| Author_xml | – sequence: 1 fullname: 山下, 太郎 organization: 熊本大学医学部附属病院 神経内科 神経難病診療体制構築事業 – sequence: 1 fullname: 安東, 由喜雄 organization: 熊本大学大学院 生命科学研究部 神経内科学分野 |
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| Copyright | 2017 公益財団法人 日本心臓財団 |
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| References | 7) Ando Y, Sekijima Y, Obayashi K, et al：Effects of tafamidis treatment on transthyretin （TTR） stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy （TTR-FAP） with Val30Met and non-Val30Met：A phase III, open-label study. J Neurol Sci 2016；362：266-271 6) Coelho T, Maia LF, Martins da Silva A, et al：Tafamidis for transthyretin familial amyloid polyneuropathy：a randomized, controlled trial. Neurology 2012；79：785-792 2) Koike H, Misu K, Ikeda S, et al：Type I （transthyretin Met30） familial amyloid polyneuropathy in Japan：early- vs late-onset form. Arch Neurol 2002；59：1771-1776 9) Merlini G, Planté-Bordeneuve V, Judge DP, et al：Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. J Cardiovasc Transl Res 2013；6：1011-1020 12) Ando Y, Tanaka Y, Ando E, et al：Effect of liver transplantation on autonomic dysfunction in familial amyloidotic polyneuropathy type I. Lancet 1995；345：195-196 10) Berk JL, Suhr OB, Obici L, et al：Repurposing diflunisal for familial amyloid polyneuropathy：a randomized clinical trial. JAMA 2013；310：2658-2667 4) Ando Y, Obayashi K, Tanaka Y, et al：Radiolabelled meta-iodobenzylguanidine in assessment of autonomic dysfunction. Lancet 1994；343：984-985 13) Yamashita T, Ando Y, Okamoto S, et al：Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology 2012；78：637-643 8) Maurer MS, Grogan DR, Judge DP, et al：Tafamidis in transthyretin amyloid cardiomyopathy：effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail 2015；8：519-526 1) Ando Y, Coelho T, Berk JL, et al：Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 2013；8：31 3) Yamashita T, Ueda M, Saga N, et al：Hereditary amyloidosis with cardiomyopathy caused by the novel variant transthyretin A36D. Amyloid 2016；23：207-208 5) Waddington Cruz M, Benson MD：A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis. Neurol Ther 2015；4：61-79 11) Sekijima Y, Tojo K, Morita H, et al：Safety and efficacy of long-term diflunisal administration in hereditary transthyretin （ATTR） amyloidosis. Amyloid 2015；22：79-83 |
| References_xml | – reference: 6) Coelho T, Maia LF, Martins da Silva A, et al：Tafamidis for transthyretin familial amyloid polyneuropathy：a randomized, controlled trial. Neurology 2012；79：785-792 – reference: 9) Merlini G, Planté-Bordeneuve V, Judge DP, et al：Effects of tafamidis on transthyretin stabilization and clinical outcomes in patients with non-Val30Met transthyretin amyloidosis. J Cardiovasc Transl Res 2013；6：1011-1020 – reference: 1) Ando Y, Coelho T, Berk JL, et al：Guideline of transthyretin-related hereditary amyloidosis for clinicians. Orphanet J Rare Dis 2013；8：31 – reference: 12) Ando Y, Tanaka Y, Ando E, et al：Effect of liver transplantation on autonomic dysfunction in familial amyloidotic polyneuropathy type I. Lancet 1995；345：195-196 – reference: 13) Yamashita T, Ando Y, Okamoto S, et al：Long-term survival after liver transplantation in patients with familial amyloid polyneuropathy. Neurology 2012；78：637-643 – reference: 7) Ando Y, Sekijima Y, Obayashi K, et al：Effects of tafamidis treatment on transthyretin （TTR） stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy （TTR-FAP） with Val30Met and non-Val30Met：A phase III, open-label study. J Neurol Sci 2016；362：266-271 – reference: 8) Maurer MS, Grogan DR, Judge DP, et al：Tafamidis in transthyretin amyloid cardiomyopathy：effects on transthyretin stabilization and clinical outcomes. Circ Heart Fail 2015；8：519-526 – reference: 2) Koike H, Misu K, Ikeda S, et al：Type I （transthyretin Met30） familial amyloid polyneuropathy in Japan：early- vs late-onset form. Arch Neurol 2002；59：1771-1776 – reference: 11) Sekijima Y, Tojo K, Morita H, et al：Safety and efficacy of long-term diflunisal administration in hereditary transthyretin （ATTR） amyloidosis. Amyloid 2015；22：79-83 – reference: 3) Yamashita T, Ueda M, Saga N, et al：Hereditary amyloidosis with cardiomyopathy caused by the novel variant transthyretin A36D. Amyloid 2016；23：207-208 – reference: 4) Ando Y, Obayashi K, Tanaka Y, et al：Radiolabelled meta-iodobenzylguanidine in assessment of autonomic dysfunction. Lancet 1994；343：984-985 – reference: 5) Waddington Cruz M, Benson MD：A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis. Neurol Ther 2015；4：61-79 – reference: 10) Berk JL, Suhr OB, Obici L, et al：Repurposing diflunisal for familial amyloid polyneuropathy：a randomized clinical trial. JAMA 2013；310：2658-2667 |
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| Title | タファミジス：遺伝性トランスサイレチンアミロイドーシスに対する蛋白質安定化剤 |
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