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Amyloid
The Journal of Protein Folding Disorders
Volume 30, 2023 - Issue 1
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Research Articles

Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial

David Adamsa Neurology Department, CHU Bicêtre, APHP, Université Paris-Saclay, Le Kremlin Bicêtre Cedex, FranceCorrespondence[email protected]
ORCID Iconhttps://orcid.org/0000-0002-8722-4108View further author information
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Ivailo L. Tournevb Department of Neurology, Clinic of Nervous Diseases, University Hospital Aleksandrovska, Medical University, Sofia, Bulgaria;c Department of Cognitive Sciences, New Bulgarian University, Sofia, BulgariaView further author information
,
Mark S. Taylord Department of Clinical Immunology and Allergy, Westmead Hospital and Westmead Clinical School, University of Sydney, Sydney, NSW, AustraliaORCID Iconhttps://orcid.org/0000-0003-1270-7209View further author information
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Teresa Coelhoe Hospital de Santo António, Centro Hospitalar Universitário do Porto, Porto, PortugalView further author information
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Violaine Planté-Bordeneuvef Neurology – Amyloid Network, CHU Henri Mondor, APHP, University Paris Est – Créteil, Créteil, FranceORCID Iconhttps://orcid.org/0000-0002-7880-2316View further author information
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John L. Berkg Boston Medical Center, Boston University, Boston, Massachusetts, USAORCID Iconhttps://orcid.org/0000-0002-1768-2373View further author information
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Alejandra González-Duarteh Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, México, D.F., MéxicoORCID Iconhttps://orcid.org/0000-0002-2434-9560View further author information
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Julian D. Gillmorei National Amyloidosis Centre, University College London, Royal Free Hospital, London, UKORCID Iconhttps://orcid.org/0000-0001-6174-9232View further author information
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Soon-Chai Lowj Department of Medicine, Division of Neurology, University Malaya Medical Centre, Kuala Lumpur, MalaysiaORCID Iconhttps://orcid.org/0000-0003-2392-6570View further author information
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Yoshiki Sekijimak Department of Medicine (Neurology & Rheumatology), Shinshu University School of Medicine, Matsumoto, JapanORCID Iconhttps://orcid.org/0000-0002-8132-2743View further author information
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Laura Obicil Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, ItalyORCID Iconhttps://orcid.org/0000-0001-7468-700XView further author information
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Chongshu Chenm Alnylam Pharmaceuticals, Cambridge, Massachusetts, USAView further author information
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Prajakta Badrim Alnylam Pharmaceuticals, Cambridge, Massachusetts, USAORCID Iconhttps://orcid.org/0000-0003-1248-616XView further author information
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Seth M. Arumm Alnylam Pharmaceuticals, Cambridge, Massachusetts, USAView further author information
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John Vestm Alnylam Pharmaceuticals, Cambridge, Massachusetts, USAView further author information
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Michael Polydefkisn Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USAORCID Iconhttps://orcid.org/0000-0002-7584-7447View further author information
ORCID Icon & The HELIOS-A Collaborators show all
Pages 18-26 | Received 28 Feb 2022, Accepted 16 Jun 2022, Published online: 23 Jul 2022

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Wenbing Zhang, Jian Ding, Wenhai Wang, Duo Wang, Yinping Pan & Dexin Xu. (2023) Status and Future Directions of Therapeutics and Prognosis of Cardiac Amyloidosis. Therapeutics and Clinical Risk Management 19, pages 581-597.
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René Rettl, Raffaella Calabretta, Franz Duca, Christina Binder, Christina Kronberger, Robin Willixhofer, Michael Poledniczek, Carolina Donà, Christian Nitsche, Dietrich Beitzke, Christian Loewe, Michaela Auer-Grumbach, Diana Bonderman, Stefan Kastl, Christian Hengstenberg, Roza Badr Eslam, Johannes Kastner, Jutta Bergler-Klein, Marcus Hacker & Andreas Kammerlander. (2023) Reduction in 99mTc-DPD myocardial uptake with therapy of ATTR cardiomyopathy. Amyloid 0:0, pages 1-10.
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Madeline Merkel, David Danese, Chongshu Chen, Jessie Wang, Aozhou Wu, Hongbo Yang & Hollis Lin. (2023) Indirect treatment comparison (ITC) of the efficacy of vutrisiran and tafamidis for hereditary transthyretin-mediated amyloidosis with polyneuropathy. Expert Opinion on Pharmacotherapy 24:10, pages 1205-1214.
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Yusuke Takahashi, Kazuhiro Oguchi, Yusuke Mochizuki, Ken Takasone, Naoki Ezawa, Akira Matsushima, Nagaaki Katoh, Masahide Yazaki & Yoshiki Sekijima. (2023) Distribution and progression of cerebral amyloid angiopathy in early-onset V30M (p.V50M) hereditary ATTR amyloidosis. Amyloid 30:1, pages 109-118.
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Articles from other publishers (24)

Jan L. De Bleecker, Kristl G. Claeys, Stéphanie Delstanche, Vinciane Van Parys, Jonathan Baets, Sébastien Tilleux & Gauthier Remiche. (2023) A retrospective survey of patients with hereditary transthyretin-mediated (hATTR) amyloidosis treated with patisiran in real-world clinical practice in Belgium. Acta Neurologica Belgica 123:3, pages 1029-1037.
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Deandre King, K. H. Vincent Lau & Michelle C. Kaku. (2023) Treatment Updates for Neuropathy in Hereditary Transthyretin Amyloidosis. Current Treatment Options in Neurology 25:6, pages 137-149.
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Melissa R. Tsoi, Jeffrey H. Lin & Ayan R. Patel. (2023) Emerging Therapies for Transthyretin Amyloidosis. Current Oncology Reports 25:6, pages 549-558.
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Maria Teresa Mallus & Vittoria Rizzello. (2023) Treatment of amyloidosis: present and future. European Heart Journal Supplements 25:Supplement_B, pages B99-B103.
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Gizem Kayki-Mutlu, Zinnet Sevval Aksoyalp, Leszek Wojnowski & Martin C. Michel. (2023) A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2022. Naunyn-Schmiedeberg's Archives of Pharmacology.
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Othman Al Musaimi, Danah Al Shaer, Fernando Albericio & Beatriz G. de la Torre. (2023) 2022 FDA TIDES (Peptides and Oligonucleotides) Harvest. Pharmaceuticals 16:3, pages 336.
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Teresa Coelho, Márcia Waddington Cruz, Chi-Chao Chao, Yeşim Parman, Jonas Wixner, Markus Weiler, Fabio A. Barroso, Noel R. Dasgupta, Shiangtung W. Jung, Eugene Schneider, Nicholas J. Viney, P. James B. Dyck, Yukio Ando, Julian D. Gillmore, Sami Khella, Morie A. Gertz, Laura Obici & John L. Berk. (2022) Characteristics of Patients with Hereditary Transthyretin Amyloidosis-Polyneuropathy (ATTRv-PN) in NEURO-TTRansform, an Open-label Phase 3 Study of Eplontersen. Neurology and Therapy 12:1, pages 267-287.
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Łukasz Wołowiec, Joanna Osiak, Anna Wołowiec, Aleksandra Wijata, Elżbieta Grześk, Mariusz Kozakiewicz, Joanna Banach, Alicja Nowaczyk, Jacek Nowaczyk & Grzegorz Grześk. (2023) Inclisiran—Safety and Effectiveness of Small Interfering RNA in Inhibition of PCSK-9. Pharmaceutics 15:2, pages 323.
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Carme Fàbrega, Anna Aviñó, Natalia Navarro, Andreia F. Jorge, Santiago Grijalvo & Ramon Eritja. (2023) Lipid and Peptide-Oligonucleotide Conjugates for Therapeutic Purposes: From Simple Hybrids to Complex Multifunctional Assemblies. Pharmaceutics 15:2, pages 320.
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Juan Jiménez-Jáimez, Eduardo Villacorta, Esther Zorio-Grima, Tomás Ripoll-Vera, Rosa Macías-Ruiz, Francisco Bermúdez-Jiménez & Javier Limeres-Freire. (2023) Selección de lo mejor del año 2022 en cardiopatías familiares y genética cardiovascular. REC: CardioClinics 58, pages S68-S73.
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Fabio Mastrocola, Frank Nunes & Paulo Sgobbi. 2023. Amyloidosis and Fabry Disease. Amyloidosis and Fabry Disease 235 276 .
Vincenzo Di Stefano, Ewan Thomas, Paolo Alonge, Valerio Giustino, Guglielmo Pillitteri, Ignazio Leale, Angelo Torrente, Antonia Pignolo, Davide Norata, Salvatore Iacono, Antonino Lupica, Antonio Palma, Giuseppe Battaglia & Filippo Brighina. (2022) Patisiran Enhances Muscle Mass after Nine Months of Treatment in ATTRv Amyloidosis: A Study with Bioelectrical Impedance Analysis and Handgrip Strength. Biomedicines 11:1, pages 62.
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C. Cristina Quarta, Marianna Fontana, Thibaud Damy, Julia Catini, Damien Simoneau, Michele Mercuri, Pablo Garcia-Pavia, Mathew S. Maurer & Giovanni Palladini. (2022) Changing paradigm in the treatment of amyloidosis: From disease-modifying drugs to anti-fibril therapy. Frontiers in Cardiovascular Medicine 9.
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Lei Zhang, Yayu Liang, Guohui Liang, Zhili Tian, Yue Zhang, Zhihui Liu & Xinying Ji. (2022) The therapeutic prospects of N-acetylgalactosamine-siRNA conjugates. Frontiers in Pharmacology 13.
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Thomas H. Brannagan3rd3rd, John L. Berk, Julian D. Gillmore, Mathew S. Maurer, Márcia Waddington‐Cruz, Marianna Fontana, Ahmad Masri, Laura Obici, Michela Brambatti, Brenda F. Baker, Lisa A. Hannan, Gustavo Buchele, Nick J. Viney, Teresa Coelho & Jose Nativi‐Nicolau. (2022) Liver‐directed drugs for t ransthyretin‐mediated amyloidosis . Journal of the Peripheral Nervous System 27:4, pages 228-237.
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Mark S. Taylor, Hasib Sidiqi, James Hare, Fiona Kwok, Bo Choi, Darren Lee, Jay Baumwol, Antonia S. Carroll, Steve Vucic, Pat Neely, Dariusz Korczyk, Liza Thomas, Peter Mollee, Graeme J. Stewart & Simon D. J. Gibbs. (2022) Current approaches to the diagnosis and management of amyloidosis. Internal Medicine Journal 52:12, pages 2046-2067.
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Noel R. Dasgupta. (2022) Care of Patients With Transthyretin Amyloidosis: the Roles of Nutrition, Supplements, Exercise, and Mental Health. The American Journal of Cardiology 185, pages S35-S42.
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Aleksandra Dondalska, Sandra Axberg Pålsson & Anna-Lena Spetz. (2022) Is There a Role for Immunoregulatory and Antiviral Oligonucleotides Acting in the Extracellular Space? A Review and Hypothesis. International Journal of Molecular Sciences 23:23, pages 14593.
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Man Amanat, Christina L. Nemeth, Amena Smith Fine, Doris G. Leung & Ali Fatemi. (2022) Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology. Pharmaceutics 14:11, pages 2389.
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Naresh Bumma, Rami Kahwash, Samir V. Parikh, Michael Isfort, Miriam Freimer, Ajay Vallakati, Elyse Redder, Courtney M. Campbell, Nidhi Sharma, Yvonne Efebera & Amro Stino. (2022) Multidisciplinary amyloidosis care in the era of personalized medicine. Frontiers in Neurology 13.
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Kosh Agarwal, James Lok & Ed Gane. (2022) Antisense oligonucleotides (ASOs) in chronic hepatitis B infection: Opportunities and challenging the orthodoxy. Journal of Hepatology 77:4, pages 906-908.
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Susan J. Keam. (2022) Vutrisiran: First Approval. Drugs 82:13, pages 1419-1425.
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