Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy

被引:0
作者
Laura Obici
Senda Ajroud-Driss
Kon-Ping Lin
John L. Berk
Julian D. Gillmore
Parag Kale
Haruki Koike
David Danese
Emre Aldinc
Chongshu Chen
John Vest
David Adams
机构
[1] IRCCS Fondazione Policlinico San Matteo,Amyloidosis Research and Treatment Centre
[2] Northwestern University Feinberg School of Medicine,Department of Neurology
[3] Taipei Veterans General Hospital,Department of Neurology
[4] Boston Medical Center,National Amyloidosis Centre, Royal Free Hospital
[5] University College London,Department of Neurology
[6] Baylor University Medical Center,Neurology Department
[7] Nagoya University Graduate School of Medicine,undefined
[8] Alnylam Pharmaceuticals,undefined
[9] Université Paris-Saclay,undefined
[10] U1195,undefined
[11] INSERM,undefined
[12] AP-HP,undefined
[13] CHU Bicêtre,undefined
关键词
ATTRv amyloidosis; hATTR amyloidosis; Nutritional status; Physical function; Polyneuropathy; Quality of life; RNA interference; Vutrisiran;
D O I
暂无
中图分类号
学科分类号
摘要
引用
收藏
页码:1759 / 1775
页数:16
相关论文
共 50 条
  • [31] Treatment response and neurofilament light chain levels with long-term patisiran in hereditary transthyretin-mediated amyloidosis with polyneuropathy: 24-month results of an open-label extension study
    Ticau, Simina
    Aldinc, Emre
    Polydefkis, Michael
    Adams, David
    Coelho, Teresa
    Ueda, Mitsuharu
    Hale, Cecilia
    Vest, John
    Nioi, Paul
    AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2024, 31 (01): : 1 - 11
  • [32] Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis
    Alejandra González-Duarte
    John L. Berk
    Dianna Quan
    Michelle L. Mauermann
    Hartmut H. Schmidt
    Michael Polydefkis
    Márcia Waddington-Cruz
    Mitsuharu Ueda
    Isabel M. Conceição
    Arnt V. Kristen
    Teresa Coelho
    Cécile A. Cauquil
    Céline Tard
    Madeline Merkel
    Emre Aldinc
    Jihong Chen
    Marianne T. Sweetser
    Jing Jing Wang
    David Adams
    Journal of Neurology, 2020, 267 : 703 - 712
  • [33] Efficacy of inotersen for neuropathic impairment scores in patients with hereditary transthyretin amyloidosis with polyneuropathy
    Yarlas, Aaron
    Lovley, Andrew
    Kessler, Asia Sikora
    Brown, Duncan
    Vera-Llonch, Montserrat
    JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, 2021, 26 (03) : 344 - 344
  • [34] Factors associated with increased health-related quality-of-life benefits in hereditary transthyretin amyloidosis polyneuropathy patients treated with inotersen
    Karam, Chafic
    Brown, Duncan
    Yang, Min
    Done, Nicolae
    Dieye, Ibou
    Bozas, Ana
    Llonch, Montserrat Vera
    Signorovitch, James
    MUSCLE & NERVE, 2022, 66 (03) : 319 - 328
  • [35] A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
    Teresa Coelho
    David Adams
    Isabel Conceição
    Márcia Waddington-Cruz
    Hartmut H. Schmidt
    Juan Buades
    Josep Campistol
    John L. Berk
    Michael Polydefkis
    Jing Jing Wang
    Jihong Chen
    Marianne T. Sweetser
    Jared Gollob
    Ole B. Suhr
    Orphanet Journal of Rare Diseases, 15
  • [36] Strategies to improve the quality of life in patients with hereditary transthyretin amyloidosis (hATTR) and autonomic neuropathy
    Thierry Gendre
    Violaine Planté-Bordeneuve
    Clinical Autonomic Research, 2019, 29 : 25 - 31
  • [37] A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis
    Coelho, Teresa
    Adams, David
    Conceicao, Isabel
    Waddington-Cruz, Marcia
    Schmidt, Hartmut H.
    Buades, Juan
    Campistol, Josep
    Berk, John L.
    Polydefkis, Michael
    Wang, Jing Jing
    Chen, Jihong
    Sweetser, Marianne T.
    Gollob, Jared
    Suhr, Ole B.
    ORPHANET JOURNAL OF RARE DISEASES, 2020, 15 (01)
  • [38] Strategies to improve the quality of life in patients with hereditary transthyretin amyloidosis (hATTR) and autonomic neuropathy
    Gendre, Thierry
    Plante-Bordeneuve, Violaine
    CLINICAL AUTONOMIC RESEARCH, 2019, 29 (Suppl 1) : 25 - 31
  • [39] A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium
    De Bleecker, Jan L.
    Claeys, Kristl G.
    Delstanche, Stephanie
    Van Parys, Vinciane
    Baets, Jonathan
    Tilleux, Sebastien
    Remiche, Gauthier
    ACTA NEUROLOGICA BELGICA, 2023, 123 (03) : 1029 - 1037
  • [40] A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium
    Jan L. De Bleecker
    Kristl G. Claeys
    Stéphanie Delstanche
    Vinciane Van Parys
    Jonathan Baets
    Sébastien Tilleux
    Gauthier Remiche
    Acta Neurologica Belgica, 2023, 123 : 1029 - 1037