Tafamidis (Fx-1006A): a first-in-class disease-modifying therapy for transthyretin familial amyloid

被引:0
|
作者
Coelho, T. [1 ]
Maia, L. [1 ]
Martins da Silva, A. [1 ]
Waddingtion-Cruz, M. [2 ]
Plante-Bordeneuve, V. [3 ]
Lozeron, P. [4 ]
Suhr, O. B. [5 ]
Campistol, J. [6 ]
Conceicao, I. M. [7 ]
Schmidt, H. [8 ]
Trigo, P. [9 ]
Packman, J. [10 ]
Grogan, D. R. [10 ]
机构
[1] Hosp Santo Antonio, Dept Neurophysiol, Oporto, Portugal
[2] Univ Fed Rio de Janeiro, Univ Hosp, Neurol Serv, BR-21941 Rio De Janeiro, Brazil
[3] CHU Henri Mondor, Dept Neurol, F-94010 Creteil, France
[4] CHU Bicetre, Dept Neurol, Le Kremlin Bicetre, France
[5] Umea Univ Hosp, Dept Publ Hlth & Clin Med, S-90185 Umea, Sweden
[6] Hosp Clin Barcelona, Renal Transplant Unit, Barcelona, Spain
[7] Hosp Santa Maria, Dept Neurol, Lisbon, Portugal
[8] Univ Klinikum Munster, Munster, Germany
[9] FLENI, Buenos Aires, DF, Argentina
[10] FoldRx Pharmaceut Inc, Cambridge, MA USA
来源
AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS | 2010年 / 17卷
关键词
D O I
暂无
中图分类号
Q5 [生物化学]; Q7 [分子生物学];
学科分类号
071010 ; 081704 ;
摘要
引用
收藏
页码:75 / 76
页数:2
相关论文
共 50 条
  • [31] Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
    Mundayat, Rajiv
    Stewart, Michelle
    Alvir, Jose
    Short, Sarah
    Ong, Moh-Lim
    Keohane, Denis
    Rill, Denise
    Sultan, Marla B.
    NEUROLOGY AND THERAPY, 2018, 7 (01) : 87 - 101
  • [32] Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS)
    Rajiv Mundayat
    Michelle Stewart
    Jose Alvir
    Sarah Short
    Moh-Lim Ong
    Denis Keohane
    Denise Rill
    Marla B. Sultan
    Neurology and Therapy, 2018, 7 : 87 - 101
  • [33] A landmark clinical trial of a novel small molecule transthyretin (TTR) stabiliser, Fx-1006A, in patients with TTR amyloid polyneuropathy:: a phase II/III, randomised, double-blind, placebo-controlled study
    Waddington-Cruz, M.
    Coelho, T.
    Maia, L.
    da Silva, A. Martins
    Plante-Bordeneuve, V.
    Suhr, O.
    Campistol, J.
    Conceicao, I.
    Schmidt, H.
    Trigo, P.
    Cros, D.
    Grogan, D. Roy
    Packman, J.
    JOURNAL OF NEUROLOGY, 2008, 255 : 107 - 107
  • [34] Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial
    Keohane, Denis
    Schwartz, Jeffrey
    Gundapaneni, Balarama
    Stewart, Michelle
    Amass, Leslie
    AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2017, 24 (01): : 30 - 36
  • [35] Discovery of the First-in-Class GSK-3β/HDAC Dual Inhibitor as Disease-Modifying Agent To Combat Alzheimer's Disease (vol 10, pg 469, 2019)
    De Simone, Angela
    La Pietra, Valeria
    Betari, Nibal
    Petragnani, Nicola
    Conte, Mariarosaria
    Daniele, Simona
    Pietrobono, Deborah
    Martini, Claudia
    Petralla, Sabrina
    Casadei, Raffaella
    Davani, Lara
    Frabetti, Flavia
    Russomanno, Pasquale
    Novellino, Ettore
    Montanari, Serena
    Tumiatti, Vincenzo
    Ballerini, Patrizia
    Sarno, Federica
    Nebbioso, Angela
    Altucci, Lucia
    Monti, Barbara
    Andrisano, Vincenza
    Milelli, Andrea
    ACS MEDICINAL CHEMISTRY LETTERS, 2019, 10 (09): : 1357 - 1357
  • [36] Ioannou A. Evolution of Disease-modifying Therapy for Transthyretin Cardiac Amyloidosis (vol 18, pg 30, 2024)
    Ioannou, Adam
    HEART INTERNATIONAL, 2024, 18 (02): : 3 - 3
  • [37] Baseline predictors of major adverse events for transthyretin amyloidosis cardiomyopathy patients treated and untreated with disease-modifying therapy
    Shahi, Karan
    Miller, Robert J. H.
    Dykstra, Steven
    Feng, Yuanchao
    Howlett, Jonathan G.
    Kassam, Shireen
    Veenhuyzen, Jan
    White, James A.
    Zepeda, Victor Jimenez
    Fine, Nowell M.
    AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2024, 31 : S87 - S88
  • [38] BASELINE PREDICTORS OF MAJOR ADVERSE EVENTS FOR TRANSTHYRETIN AMYLOIDOSIS CARDIOMYOPATHY PATIENTS TREATED AND UNTREATED WITH DISEASE-MODIFYING THERAPY
    Shahi, Karan
    Miller, Robert J. H.
    Dykstra, Steven
    Feng, Yuanchao
    Howlett, Jonathan G.
    Kassam, Shireen
    Veenhuyzen, Jan
    White, James
    Fine, Nowell Mark
    JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY, 2024, 83 (13) : 507 - 507
  • [39] What prompts a switch in disease-modifying therapy beyond first-line therapies?
    Salter, A.
    Tyry, T.
    Belletti, D.
    Malhotra, M.
    Marrie, R.
    Fox, R.
    Cutter, G.
    JOURNAL OF NEUROLOGY, 2012, 259 : S188 - S188
  • [40] Discontinuation of first-line disease-modifying therapy in relapse onset multiple sclerosis
    Coerver, E. M. E.
    Bourass, A.
    Wessels, M. H. J.
    van Kempen, Z. L. E.
    Jasperse, M. M. S.
    Tonino, B. A. R.
    Barkhof, F.
    Mostert, J.
    Uitdehaag, B. M. J.
    Killestein, J.
    Strijbis, E. M. M.
    MULTIPLE SCLEROSIS AND RELATED DISORDERS, 2023, 74