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
来源：
Neurology and Therapy | 2023年 / 12卷 / 5期
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
  • [41] mNIS+7and lower limb function in inotersen treatment ofhereditary transthyretin-mediated amyloidosis
    Dyck, P. James B.
    Kincaid, John C.
    Wiesman, Janice F.
    Polydefkis, Michael
    Litchy, William J.
    Mauermann, Michelle L.
    Ackermann, Elizabeth J.
    Guthrie, Spencer
    Pollock, Michael
    Jung, Shiangtung W.
    Baker, Brenda F.
    Dyck, Peter J.
    MUSCLE & NERVE, 2020, 62 (04) : 502 - 508
  • [42] Patisiran Pharmacokinetics, Pharmacodynamics, and Exposure-Response Analyses in the Phase 3 APOLLO Trial in Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis
    Zhang, Xiaoping
    Goel, Varun
    Attarwala, Husain
    Sweetser, Marianne T.
    Clausen, Valerie A.
    Robbie, Gabriel J.
    JOURNAL OF CLINICAL PHARMACOLOGY, 2020, 60 (01) : 37 - 49
  • [43] Clinical 3-D Gait Assessment of Patients With Polyneuropathy Associated With Hereditary Transthyretin Amyloidosis
    Vilas-Boas, Maria do Carmo
    Rocha, Ana Patricia
    Cardoso, Marcio Neves
    Fernandes, Jose Maria
    Coelho, Teresa
    Cunha, Joao Paulo Silva
    FRONTIERS IN NEUROLOGY, 2020, 11
  • [44] Evaluating Prognostic Factors for Liver Transplantation Among United States Patients With Hereditary Transthyretin-Mediated (hATTR) Amyloidosis Using National Registry Data
    Brandman, Danielle
    Lin, Hollis
    McManus, Anastasia
    Agarwal, Sonalee
    Gache, Larry M.
    Irish, William
    Gollob, Jared
    Zivkovic, Sasa A.
    PROGRESS IN TRANSPLANTATION, 2019, 29 (03) : 213 - 219
  • [45] Effect of Eplontersen on Cardiac Structure and Function in Patients With Hereditary Transthyretin Amyloidosis
    Masri, Ahmad
    Maurer, Mathew S.
    Claggett, Brian L.
    Kulac, Ian
    Cruz, Marcia Waddington
    Concei, Isabel
    Weiler, Markus
    Berk, John L.
    Gertz, Morie
    Gillmore, Julian D.
    Rush, Stephen
    Chen, Jersey
    Zhou, Wunan
    Kwoh, Jesse
    Duran, Jason M.
    Tsimikas, Sotirios
    Solomon, Scott D.
    JOURNAL OF CARDIAC FAILURE, 2024, 30 (08) : 973 - 980
  • [46] Hereditary Transthyretin Amyloidosis and the Impact of Classic and New Treatments on Kidney Function: A Review
    Melendrez-Balcazar, Evelyn
    Aranda-Vela, Karla
    Cervantes-Hernandez, Alberto
    Lopez-Cureno, Samuel
    AMERICAN JOURNAL OF KIDNEY DISEASES, 2024, 84 (02) : 224 - 231
  • [47] Exploring Cardiac Sympathetic Denervation in Transthyretin-Mediated Hereditary Amyloidosis (ATTRv): Insights from 123I-mIBG Scintigraphy
    De Feo, Maria Silvia
    Cambieri, Chiara
    Galosi, Eleonora
    Frantellizzi, Viviana
    Chimenti, Cristina
    Luigetti, Marco
    Sciarrone, Maria Ausilia
    Graziani, Francesca
    Leonardi, Luca
    Musumeci, Beatrice
    Libonati, Laura
    Moret, Federica
    D'Andrea, Edoardo
    Di Giulio, Matteo
    Garibaldi, Matteo
    Forcina, Francesca
    Truini, Andrea
    De Vincentis, Giuseppe
    Inghilleri, Maurizio
    Ceccanti, Marco
    DIAGNOSTICS, 2025, 15 (04)
  • [48] Effects of eplontersen on symptoms of autonomic neuropathy in hereditary transthyretin-mediated amyloidosis: secondary analysis from the NEURO-TTRansform trial
    Wixner, Jonas
    Berk, John L.
    Adams, David
    Polydefkis, Michael
    Conceicao, Isabel
    Attarian, Shahram
    Gillmore, Julian D.
    Dyck, P. James B.
    Folkvaljon, Folke
    Zhou, Wunan
    Chen, Jersey
    Viney, Nicholas J.
    Kwoh, T. Jesse
    Coelho, Teresa
    Waddington-Cruz, Marcia
    AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2025, 32 (01): : 29 - 38
  • [49] Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression
    Conceicao, Isabel
    Coelho, Teresa
    Rapezzi, Claudio
    Parman, Yesim
    Obici, Laura
    Galan, Lucia
    Rousseau, Antoine
    AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, 2019, 26 (03): : 103 - 111
  • [50] Nonclinical Safety Profile of Revusiran, a 1st-Generation GalNAc-siRNA Conjugate for Treatment of Hereditary Transthyretin-Mediated Amyloidosis
    Sutherland, Jessica E.
    Hettinger, Julia L.
    Chan, Amy
    Gilbert, Jason
    Warner, Garvin L.
    Davis, Wendell P.
    NUCLEIC ACID THERAPEUTICS, 2020, 30 (01) : 33 - 49
12345