Literature DB >> 22378262

Tafamidis.

Gerard Said, Seden Grippon, Peter Kirkpatrick.   

Abstract

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.

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Year:  2012        PMID： 22378262     DOI： 10.1038/nrd3675

Source DB:  PubMed          Journal:  Nat Rev Drug Discov        ISSN： 1474-1776            Impact factor:   84.694


  6 in total

1.  Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action.

Authors:  Hossein Razavi; Satheesh K Palaninathan; Evan T Powers; R Luke Wiseman; Hans E Purkey; Nilofar N Mohamedmohaideen; Songpon Deechongkit; Kyle P Chiang; Maria T A Dendle; James C Sacchettini; Jeffery W Kelly
Journal:  Angew Chem Int Ed Engl       Date:  2003-06-23       Impact factor: 15.336

Review 2.  The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug.

Authors:  Steven M Johnson; Stephen Connelly; Colleen Fearns; Evan T Powers; Jeffery W Kelly
Journal:  J Mol Biol       Date:  2012-01-05       Impact factor: 5.469

3.  Manifestations of transthyretin-related familial amyloidotic polyneuropathy: long-term follow-up of Japanese patients after liver transplantation.

Authors:  Yuki Ohya; Sadahisa Okamoto; Masayoshi Tasaki; Mitsuharu Ueda; Hirofumi Jono; Konen Obayashi; Kazuhisa Takeda; Hideaki Okajima; Katsuhiro Asonuma; Ryuhei Hara; Hidenobu Tanihara; Yukio Ando; Yukihiro Inomata
Journal:  Surg Today       Date:  2011-08-26       Impact factor: 2.549

4.  Inhibiting transthyretin amyloid fibril formation via protein stabilization.

Authors:  G J Miroy; Z Lai; H A Lashuel; S A Peterson; C Strang; J W Kelly
Journal:  Proc Natl Acad Sci U S A       Date:  1996-12-24       Impact factor: 11.205

Review 5.  Familial amyloid polyneuropathy.

Authors:  Violaine Planté-Bordeneuve; Gerard Said
Journal:  Lancet Neurol       Date:  2011-12       Impact factor: 44.182

6.  Heterogeneity of penetrance in familial amyloid polyneuropathy, ATTR Val30Met, in the Swedish population.

Authors:  Urban Hellman; Flora Alarcon; Hans-Erik Lundgren; Ole B Suhr; Catherine Bonaiti-Pellié; Violaine Planté-Bordeneuve
Journal:  Amyloid       Date:  2008-09       Impact factor: 7.141
  6 in total
  23 in total

Review 1.  Disease-modifying therapy for proteinopathies: Can the exception become the rule?

Authors:  Gal Bitan
Journal:  Prog Mol Biol Transl Sci       Date:  2019-08-07       Impact factor: 3.622

Review 2.  Cardiac Amyloidosis: Diagnosis and Treatment Strategies.

Authors:  Mirela Tuzovic; Eric H Yang; Arnold S Baas; Eugene C Depasquale; Mario C Deng; Daniel Cruz; Gabriel Vorobiof
Journal:  Curr Oncol Rep       Date:  2017-07       Impact factor: 5.075

Review 3.  [Genetics of neuropathies].

Authors:  B Gess; A Schirmacher; P Young
Journal:  Nervenarzt       Date:  2013-02       Impact factor: 1.214

4.  Novel Antibody for the Treatment of Transthyretin Amyloidosis.

Authors:  Akihiko Hosoi; Yu Su; Masaharu Torikai; Hirofumi Jono; Daisuke Ishikawa; Kenji Soejima; Hirofumi Higuchi; Jianying Guo; Mitsuharu Ueda; Genki Suenaga; Hiroaki Motokawa; Tokunori Ikeda; Satoru Senju; Toshihiro Nakashima; Yukio Ando
Journal:  J Biol Chem       Date:  2016-10-07       Impact factor: 5.157

5.  Diagnostic hallmarks and pitfalls in late-onset progressive transthyretin-related amyloid-neuropathy.

Authors:  Maike F Dohrn; Christoph Röcken; Jan L De Bleecker; Jean-Jacques Martin; Matthias Vorgerd; Peter Y Van den Bergh; Andreas Ferbert; Katrin Hinderhofer; J Michael Schröder; Joachim Weis; Jörg B Schulz; Kristl G Claeys
Journal:  J Neurol       Date:  2013-10-08       Impact factor: 4.849

6.  Left ventricular amyloid deposition in patients with heart failure and preserved ejection fraction.

Authors:  Selma F Mohammed; Sultan A Mirzoyev; William D Edwards; Ahmet Dogan; Donna R Grogan; Shannon M Dunlay; Veronique L Roger; Morie A Gertz; Angela Dispenzieri; Steven R Zeldenrust; Margaret M Redfield
Journal:  JACC Heart Fail       Date:  2014-04       Impact factor: 12.035

7.  Neurophysiological markers of small fibre neuropathy in TTR-FAP mutation carriers.

Authors:  Jean-Pascal Lefaucheur; Sophie Ng Wing Tin; Philippe Kerschen; Thibaud Damy; Violaine Planté-Bordeneuve
Journal:  J Neurol       Date:  2013-01-11       Impact factor: 4.849

Review 8.  The Amyloid Phenomenon and Its Significance in Biology and Medicine.

Authors:  Christopher M Dobson; Tuomas P J Knowles; Michele Vendruscolo
Journal:  Cold Spring Harb Perspect Biol       Date:  2020-02-03       Impact factor: 10.005

9.  Use of Drugs for ATTRv Amyloidosis in the Real World: How Therapy Is Changing Survival in a Non-Endemic Area.

Authors:  Massimo Russo; Luca Gentile; Vincenzo Di Stefano; Gianluca Di Bella; Fabio Minutoli; Antonio Toscano; Filippo Brighina; Giuseppe Vita; Anna Mazzeo
Journal:  Brain Sci       Date:  2021-04-27

Review 10.  Guideline of transthyretin-related hereditary amyloidosis for clinicians.

Authors:  Yukio Ando; Teresa Coelho; John L Berk; Márcia Waddington Cruz; Bo-Göran Ericzon; Shu-ichi Ikeda; W David Lewis; Laura Obici; Violaine Planté-Bordeneuve; Claudio Rapezzi; Gerard Said; Fabrizio Salvi
Journal:  Orphanet J Rare Dis       Date:  2013-02-20       Impact factor: 4.123
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