{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,19]],"date-time":"2026-10-19T22:51:25Z","timestamp":1792450285179,"version":"4.5.0"},"reference-count":26,"publisher":"Massachusetts Medical Society","issue":"1","license":[{"start":{"date-parts":[[2018,7,5]],"date-time":"2018-07-05T00:00:00Z","timestamp":1530748800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/www.nejmgroup.org\/legal\/terms-of-use.htm"}],"funder":[{"DOI":"10.13039\/100006400","name":"Alnylam Pharmaceuticals","doi-asserted-by":"publisher","award":["Not applicable"],"award-info":[{"award-number":["Not applicable"]}],"id":[{"id":"10.13039\/100006400","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["N Engl J Med"],"published-print":{"date-parts":[[2018,7,5]]},"DOI":"10.1056\/nejmoa1716153","type":"journal-article","created":{"date-parts":[[2018,7,4]],"date-time":"2018-07-04T17:01:16Z","timestamp":1530723676000},"page":"11-21","source":"Crossref","is-referenced-by-count":2934,"title":["Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis"],"prefix":"10.1056","volume":"379","author":[{"given":"David","family":"Adams","sequence":"first","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alejandra","family":"Gonzalez-Duarte","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William D.","family":"O\u2019Riordan","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chih-Chao","family":"Yang","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mitsuharu","family":"Ueda","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arnt V.","family":"Kristen","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ivailo","family":"Tournev","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hartmut H.","family":"Schmidt","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teresa","family":"Coelho","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; 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the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeeyoung","family":"Oh","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; the National Institute of Medical Sciences and Nutrition\u2013Salvador..."}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yesim","family":"Parman","sequence":"additional","affiliation":[{"name":"From Assistance Publique\u2013H\u00f4pitaux de Paris (APHP), National Reference Center for Familial Amyloidotic Polyneuropathy, Centre Hospitalier Universitaire (CHU) Bic\u00eatre, INSERM Unit\u00e9 1195, Universit\u00e9 Paris-Sud, Le Kremlin-Bic\u00eatre (D.A.), the Department of Neuromuscular Disorders and ALS, H\u00f4pital de la Timone, Marseille (S.A.), and the Department of Neurology, Amyloid Network, CHU Henri Mondor\u2013APHP, Cr\u00e9teil (V.P.-B.) \u2014 all in France; 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