Tafamidis

被引:74
作者
Said, Gerard [1 ]
Grippon, Seden [2 ]
Kirkpatrick, Peter
机构
[1] Univ Pitie Salpetriere, Ctr Hosp, Dept Neurol, F-75651 Paris 13, France
[2] IMS Hlth, London NW1 6JB, England
关键词
FAMILIAL AMYLOID POLYNEUROPATHY;
D O I
10.1038/nrd3675
中图分类号
Q81 [生物工程学(生物技术)]; Q93 [微生物学];
学科分类号
071005 ; 0836 ; 090102 ; 100705 ;
摘要
In November 2011, tafamidis (Vyndaqel; Pfizer), a small molecule that inhibits the dissociation of transthyretin tetramers, was granted marketing authorization by the European Commission for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurological impairment. © 2012 Macmillan Publishers Limited. All rights reserved.
引用
收藏
页码:185 / 186
页数:2
相关论文
共 7 条
[1]  
European Medicines Agency (EMA), 2011, EUR PUBL ASS REP
[2]   Heterogeneity of penetrance in familial amyloid polyneuropathy, ATTR Val30Met, in the Swedish population [J].
Hellman, Urban ;
Alarcon, Flora ;
Lundgren, Hans-Erik ;
Suhr, Ole B. ;
Bonaiti-Pellie, Catherine ;
Plante-Bordeneuve, Violaine .
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2008, 15 (03) :181-186
[3]   The Transthyretin Amyloidoses: From Delineating the Molecular Mechanism of Aggregation Linked to Pathology to a Regulatory-Agency-Approved Drug [J].
Johnson, Steven M. ;
Connelly, Stephen ;
Fearns, Colleen ;
Powers, Evan T. ;
Kelly, Jeffery W. .
JOURNAL OF MOLECULAR BIOLOGY, 2012, 421 (2-3) :185-203
[4]   Inhibiting transthyretin amyloid fibril formation via protein stabilization [J].
Miroy, GJ ;
Lai, ZH ;
Lashuel, HA ;
Peterson, SA ;
Strang, C ;
Kelly, JW .
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA, 1996, 93 (26) :15051-15056
[5]   Manifestations of transthyretin-related familial amyloidotic polyneuropathy: Long-term follow-up of Japanese patients after liver transplantation [J].
Ohya, Yuki ;
Okamoto, Sadahisa ;
Tasaki, Masayoshi ;
Ueda, Mitsuharu ;
Jono, Hirofumi ;
Obayashi, Konen ;
Takeda, Kazuhisa ;
Okajima, Hideaki ;
Asonuma, Katsuhiro ;
Hara, Ryuhei ;
Tanihara, Hidenobu ;
Ando, Yukio ;
Inomata, Yukihiro .
SURGERY TODAY, 2011, 41 (09) :1211-1218
[6]   Familial amyloid polyneuropathy [J].
Plante-Bordeneuve, Violaine ;
Said, Gerard .
LANCET NEUROLOGY, 2011, 10 (12) :1086-1097
[7]   Benzoxazoles as transthyretin amyloid fibril inhibitors: Synthesis, evaluation, and mechanism of action [J].
Razavi, H ;
Palaninathan, SK ;
Powers, ET ;
Wiseman, RL ;
Purkey, HE ;
Mohamedmohaideen, NN ;
Deechongkit, S ;
Chiang, KP ;
Dendle, MTA ;
Sacchettini, JC ;
Kelly, JW .
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION, 2003, 42 (24) :2758-2761