Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy,The New England Journal of Medicine

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Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy
The New England Journal of Medicine ( IF 96.2 ) Pub Date : 2024-08-30 , DOI: 10.1056/nejmoa2409134
Marianna Fontana ₁ , John L Berk ₁ , Julian D Gillmore ₁ , Ronald M Witteles ₁ , Martha Grogan ₁ , Brian Drachman ₁ , Thibaud Damy ₁ , Pablo Garcia-Pavia ₁ , Jorg Taubel ₁ , Scott D Solomon ₁ , Farooq H Sheikh ₁ , Nobuhiro Tahara ₁ , José González-Costello ₁ , Kenichi Tsujita ₁ , Caroline Morbach ₁ , Zoltán Pozsonyi ₁ , Mark C Petrie ₁ , Diego Delgado ₁ , Peter Van der Meer ₁ , Andrew Jabbour ₁ , Antoine Bondue ₁ , Darae Kim ₁ , Olga Azevedo ₁ , Steen Hvitfeldt Poulsen ₁ , Ali Yilmaz ₁ , Ewa A Jankowska ₁ , Vincent Algalarrondo ₁ , Andrew Slugg ₁ , Pushkal P Garg ₁ , Katherine L Boyle ₁ , Elena Yureneva ₁ , Nancy Silliman ₁ , Lilli Yang ₁ , Jihong Chen ₁ , Satish A Eraly ₁ , John Vest ₁ , Mathew S Maurer ₁ ,

Affiliation

From the National Amyloidosis Centre, Division of Medicine, University College London, Royal Free Hospital (M.F., J.D.G.), and Richmond Pharmacology (J.T.), London, and the Institute of Cardiovascular and Medical Sciences, British Heart Foundation Glasgow Cardiovascular Research Centre, Glasgow (M.C.P.) - all in the United Kingdom; Boston University School of Medicine (J.L.B.) and the Cardiovascular Division, Brigham and Women's Hospital (S.D.S.) - both in Boston; the Division of Cardiovascular Medicine, Stanford University School of Medicine, Stanford, CA (R.M.W.); the Department of Cardiovascular Diseases, Mayo Clinic College of Medicine, Rochester, MN (M.G.); the Division of Cardiology, Penn Presbyterian Medical Center, University of Pennsylvania Health System, Philadelphia (B.D.); the Cardiology Department and French National Reference Centre for Cardiac Amyloidosis, GRC Amyloid Research Institute and Clinical Investigation Centre 1430 at Hôpitaux Universitaires Henri-Mondor, Assistance Publique-Hôpitaux de Paris (AP-HP), and Institut Mondor de Recherche Biomédicale, INSERM, Université Paris Est Creteil, Creteil (T.D.), and the Department of Cardiology, French National Reference Center for Cardiac Amyloidosis, Bichat University Hospital, AP-HP, Paris (V.A.) - all in France; the Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Health Research Institute of the Puerta de Hierro Majadahonda-Segovia, Centro de Investigación Biomédica en Red Enfermedades Cardiovasculares (CIBER-CV), and Centro Nacional de Investigaciones Cardiovasculares (P.G.-P.), and CIBER-CV (J.G.-C.), Madrid, and the Department of Cardiology, Hospital Universitari de Bellvitge, Instituto de Investigación Biomédica de Bellvitge, and Universitat de Barcelona, Barcelona (J.G.-C.) - all in Spain; MedStar Heart and Vascular Institute, MedStar Health, and Georgetown University School of Medicine - both in Washington, DC (F.H.S.); the Division of Cardiovascular Medicine, Department of Medicine, Kurume University School of Medicine, Kurume (N.T.), and the Department of Cardiovascular Medicine, Graduate School of Medical Sciences, Kumamoto University, Kumamoto (K.T.) - both in Japan; the Department of Clinical Research and Epidemiology, Comprehensive Heart Failure Center, University Hospital Würzburg, and the Department of Medicine I, University Hospital Würzburg, Würzburg (C.M.), and the Division of Cardiovascular Imaging, University Hospital Münster, Münster (A.Y.) - both in Germany; the Department of Internal Medicine and Hematology, Semmelweis University, Budapest, Hungary (Z.P.); the Department of Cardiology, University Health Network of Toronto, Toronto (D.D.); University Medical Center Groningen, University of Groningen, Groningen, the Netherlands (P.V.M.); the Victor Chang Cardiac Research Institute, the Cardiology Department, St. Vincent's Hospital, and the School of Clinical Medicine, University of New South Wales - all in Sydney (A.J.); the Department of Cardiology, Hôpital Universitaire de Bruxelles-Hôpital Erasme, Université Libre de Bruxelles, Brussels (A.B.); the Department of Medicine, Heart Vascular Stroke Institute, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, South Korea (D.K.); the Cardiology Department, Hospital Senhora da Oliveira-Guimarães, Guimarães, and the School of Medicine, University of Minho, Braga - both in Portugal (O.A.); the Department of Cardiology, Aarhus University Hospital, Aarhus, Denmark (S.H.P.); the Division of Translational Cardiology and Clinical Registries, Institute of Heart Diseases, Wrocław Medical University, Wrocław, Poland (E.A.J.); Alnylam Pharmaceuticals, Cambridge, MA (A.S., P.P.G., K.L.B., E.Y., N.S., L.Y., J.C., S.A.E., J.V.); and Columbia University Irving Medical Center, New York (M.S.M.).

Transthyretin amyloidosis with cardiomyopathy (ATTR-CM) is a progressive, fatal disease. Vutrisiran, a subcutaneously administered RNA interference therapeutic agent, inhibits the production of hep...

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