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

Author:

Adams David1ORCID,Tournev Ivailo L.23,Taylor Mark S.4ORCID,Coelho Teresa5,Planté-Bordeneuve Violaine6ORCID,Berk John L.7ORCID,González-Duarte Alejandra8ORCID,Gillmore Julian D.9ORCID,Low Soon-Chai10ORCID,Sekijima Yoshiki11ORCID,Obici Laura12ORCID,Chen Chongshu13,Badri Prajakta13ORCID,Arum Seth M.13,Vest John13,Polydefkis Michael14ORCID,

Affiliation:

1. Neurology Department, CHU Bicêtre, APHP, Université Paris-Saclay, Le Kremlin Bicêtre Cedex, France

2. Department of Neurology, Clinic of Nervous Diseases, University Hospital Aleksandrovska, Medical University, Sofia, Bulgaria

3. Department of Cognitive Sciences, New Bulgarian University, Sofia, Bulgaria

4. Department of Clinical Immunology and Allergy, Westmead Hospital and Westmead Clinical School, University of Sydney, Sydney, NSW, Australia

5. Hospital de Santo António, Centro Hospitalar Universitário do Porto, Porto, Portugal

6. Neurology – Amyloid Network, CHU Henri Mondor, APHP, University Paris Est – Créteil, Créteil, France

7. Boston Medical Center, Boston University, Boston, Massachusetts, USA

8. Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, México, D.F., México

9. National Amyloidosis Centre, University College London, Royal Free Hospital, London, UK

10. Department of Medicine, Division of Neurology, University Malaya Medical Centre, Kuala Lumpur, Malaysia

11. Department of Medicine (Neurology & Rheumatology), Shinshu University School of Medicine, Matsumoto, Japan

12. Amyloidosis Research and Treatment Centre, IRCCS Fondazione Policlinico San Matteo, Pavia, Italy

13. Alnylam Pharmaceuticals, Cambridge, Massachusetts, USA

14. Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland, USA

Funder

Alnylam Pharmaceuticals

Publisher

Informa UK Limited

Subject

Internal Medicine

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