Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

New England Journal of Medicine - Tập 379 Số 1 - Trang 11-21 - 2018
David Adams1, Alejandra González‐Duarte2, William O’Riordan3, Chih‐Chao Yang4, Mitsuharu Ueda5, Arnt V. Kristen6, Ivailo Tournev7, Hartmut Schmidt8, Teresa Coelho9, John L. Berk10, Kon-Ping Lin11, Giuseppe Vita12, Shahram Attarian13, Violaine Planté‐Bordeneuve14, Michelle M. Mezei15, Josep M. Campistol16, Juan Buades17, Thomas H. Brannagan18, Byoung J. Kim19, Jeeyoung Oh20, Yeşim Parman21, Yoshiki Sekijima22, Philip N. Hawkins23, Scott D. Solomon24, Michael Polydefkis25, P. James B. Dyck26, Pritesh J. Gandhi27, Sunita Goyal27, Jihong Chen27, Andrew Strahs27, Saraswathy V. Nochur27, Marianne T. Sweetser27, Pushkal Garg27, Akshay Vaishnaw27, Jared Gollob27, Ole B. Suhr28
1Bicêtre, INSERM Unité 1195, Université Paris-Sud, Le Kremlin-Bicêtre
2National Institute of Medical Sciences and Nutrition–Salvador Zubiran, Mexico City
3Department of Clinical Research, eStudySite, San Diego, CA
4Department of Neurology, National Taiwan University Hospital
5Kumamoto University Hospital, Kumamoto, Taipei, Taiwan
6Department of Cardiology, University of Heidelberg, Heidelberg
7University Multiprofile Hospital for Active Treatment, Sofia, Bulgaria
8Medizinische Klinik B für Gastroenterologie und Hepatologie, Universitätsklinikum Münster, Münster
9Hospital de Santo António, Centro Hospitalar do Porto, Porto, Portugal
10Amyloidosis Center, Department of Medicine, Boston Medical Center
11Department of Neurology, Taipei Veterans General Hospital
12Department of Clinical and Experimental Medicine, University Hospital of Messina, Messina, Italy
13Department of Neuromuscular Disorders and ALS, Hôpital de la Timone, Marseille
14Department of Neurology, Amyloid Network, CHU Henri Mondor–APHP, Créteil
15Departments of Neurology and Medicine, Vancouver General Hospital, Vancouver, BC, Canada
16Department of Nephrology, Hospital Clinic, Barcelona
17Balearic Islands Health Research Institute and Hospital Son Llatzer, Palma de Mallorca
18Department of Neurology, Columbia University, College of Physicians and Surgeons, New York, Boston
19Samsung Medical Center, Sungkyunkwan University School of Medicine, Boston
20Department of Neurology, Konkuk University Medical Center, Boston
21Department of Neurology, Istanbul University, Istanbul, Turkey
22Shinshu University School of Medicine, Matsumoto, Taipei, Taiwan
23Division of Medicine, University College London, London
24Harvard Medical School
25Johns Hopkins Bayview Medical Center, Baltimore
26Department of Neurology, Mayo Clinic, Rochester, MN
27Alnylam Pharmaceuticals, Cambridge, MA
28Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden

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New England Journal of Medicine

Tập 379 Số 1

11-21

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