Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Varl30Met: A phase III, open-label study

Yukio Ando; Yoshiki Sekijima; Konen Obayashi; Taro Yamashita; Mitsuharu Ueda; Yohei Misumi; Hiroshi Morita; Katsuyuki Machii; Makoto Ohta; Ami Takata; Shu-ichi Ikeda

首页> 外文期刊>Journal of the Neurological Sciences: Official Bulletin of the World Federation of Neurology >Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Varl30Met: A phase III, open-label study

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Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Varl30Met: A phase III, open-label study

机译：TAFAMIDIS治疗对日本家族淀粉样蛋白（TTR-FAP）患者进行TRANSTHYRETIN（TTR）稳定，疗效和安全性的影响，具有VAL30MET和非VARL30MET：III期，开放标签研究

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Introduction: The efficacy and safety of tafamidis in transthyretin (TTR) familial amyloid polyneuropathy (TTR-FAP) were evaluated in this open-label study.

机译：简介：在该开放标签研究中评估了TARANTHYRETIN（TTR）家族性淀粉样蛋白多肌病（TTR-FAP）的TAFAMIDIS的疗效和安全性。

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来源
《Journal of the Neurological Sciences: Official Bulletin of the World Federation of Neurology》 |2016年第1期|共6页
作者
Yukio Ando; Yoshiki Sekijima; Konen Obayashi; Taro Yamashita; Mitsuharu Ueda; Yohei Misumi; Hiroshi Morita; Katsuyuki Machii; Makoto Ohta; Ami Takata; Shu-ichi Ikeda;
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作者单位

Kumamoto Univ Grad Sch Med Sci Dept Neurol Kumamoto 8608556 Japan;

Shinshu Univ Sch Med Dept Med Neurol &

Rheumatol Matsumoto Nagano 390 Japan;

Kumamoto Univ Grad Sch Hlth Sci Dept Morphol &

Physiol Sci Kumamoto Japan;

Kumamoto Univ Grad Sch Med Sci Dept Neurol Kumamoto 8608556 Japan;

Kumamoto Univ Grad Sch Med Sci Dept Neurol Kumamoto 8608556 Japan;

Kumamoto Univ Grad Sch Med Sci Dept Neurol Kumamoto 8608556 Japan;

Shinshu Univ Sch Med Dept Med Neurol &

Rheumatol Matsumoto Nagano 390 Japan;

Pfizer Japan Inc Tokyo Japan;

Pfizer Japan Inc Tokyo Japan;

Pfizer Japan Inc Tokyo Japan;

Shinshu Univ Sch Med Dept Med Neurol &

Rheumatol Matsumoto Nagano 390 Japan;

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原文格式 PDF
正文语种 eng
中图分类神经病学;
关键词
Amyloid; familial amyloid polyneuropathy; tafamidis; transthyretin amyloidosis; TTR Val30Met; TTR non-Val30Met;

机译：淀粉样蛋白;家族性淀粉样蛋白多肌病;Tafamidis;Transthyretin淀粉样蛋白病;TTR Val30met;TTR非Val30met;

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1. Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Varl30Met: A phase III, open-label study [J] . Yukio Ando, Yoshiki Sekijima, Konen Obayashi, Journal of the Neurological Sciences: Official Bulletin of the World Federation of Neurology . 2016,第1期

机译：TAFAMIDIS治疗对日本家族淀粉样蛋白（TTR-FAP）患者进行TRANSTHYRETIN（TTR）稳定，疗效和安全性的影响，具有VAL30MET和非VARL30MET：III期，开放标签研究
2. Transthyretin-related Familial Amyloid Polyneuropathy (TTR-FAP) caused by a very rare, de novo mutation in a Polish patient. [J] . Marta Lipowska, Dorota Rowczenio, Janet Gilbertson, Orphanet journal of rare diseases . 2015,第S1期

机译：运甲状腺素蛋白相关的家族性淀粉样变性神经病（TTR-FAP），是由波兰患者中非常罕见的从头突变引起的。
3. Genotypic and phenotypic presentation of transthyretin-related familial amyloid polyneuropathy (TTR-FAP) in Turkey [J] . Durmus-Tekce Hacer, Matur Zeliha, Atmaca Murat Mert, Neuromuscular disorders: NMD . 2016,第7期

机译：运甲状腺素蛋白相关的家族性淀粉样蛋白多神经病（TTR-FAP）在土耳其的基因型和表型表现
4. Safety and Efficacy of the Immunomodulatory Drug (IMiD) Lenalidomide in Patients with Lymphoma: Development of RU051417I - Phase I/II Open-Label Study of R-ICE (Rituximab-Ifosfamide-Carboplatin-Etoposide) with Lenalidomide [R2-ICE] in Patients with First-Relapse/Primary Refractory Diffuse Large B-Cell Lymphoma (DLBCL). [D] . Kasi, Pashtoon Murtaza. 2018

机译：免疫调节药物来那度胺在淋巴瘤患者中的安全性和有效性：RU051417I-R-ICE（利妥昔单抗-异环磷酰胺-卡铂-依托泊苷）与来那度胺[R2-ICE]的I / II期开放标签研究的进展初次复发/原发性难治性弥漫性大B细胞淋巴瘤（DLBCL）。
5. Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe: where are we now? A European network approach to defining the epidemiology and management patterns for TTR-FAP [O] . Yesim Parman, David Adams, Laura Obici, -1

机译：在欧洲的甲状腺素转运蛋白家族性淀粉样变性神经病（TTR-FAP）已有60年了：我们现在在哪里？欧洲网络方法用于定义TTR-FAP的流行病学和管理模式
6. Transthyretin-related Familial Amyloid Polyneuropathy (TTR-FAP) caused by a very rare, de novo mutation in a Polish patient. [O] . Marta Lipowska, Dorota Rowczenio, Janet Gilbertson, 2015

机译：在波兰患者中由非常罕见的De Novo突变引起的Transthyretin相关的家族性淀粉样蛋白多变疗法（TTR-FAP）。

Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Varl30Met: A phase III, open-label study

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