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Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.

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Title: Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.
Authors: Adams D; Department of Neurology, Centre Hospitalo Universitaire Bicêtre, Assistance Publique Hopitaux de Paris, Université Paris-Saclay, L'Institut national de la santé et de la recherche médicale U-1195, Le Kremlin Bicêtre Cedex, France., Wixner J; Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden., Polydefkis M; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland., Berk JL; Boston University School of Medicine, Boston, Massachusetts., Conceição IM; Department of Neurosciences and Mental Health, Unidade Local De Saúde Santa Maria, Hospital de Santa Maria, Centro Académico de Medicina de Lisboa, Lisbon, Portugal.; Instituto de Fisiologia, Instituto de Medicina Molecular, Centro de Estudos Egas Moniz, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal., Dispenzieri A; Division of Hematology, Mayo Clinic, Rochester, Minnesota., Peltier A; Department of Neurology, Vanderbilt University Medical Center, Nashville, Tennessee., Ueda M; Department of Neurology, Kumamoto University Hospital, Kumamoto, Japan., Bender S; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Capocelli K; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Jay PY; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Yureneva E; Alnylam Pharmaceuticals, Cambridge, Massachusetts., Obici L; Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
Corporate Authors: patisiran Global OLE study group
Source: JAMA neurology [JAMA Neurol] 2025 Mar 01; Vol. 82 (3), pp. 228-236.
Publication Type: Journal Article; Randomized Controlled Trial; Multicenter Study; Clinical Trial, Phase II
Journal Info: Publisher: American Medical Association Country of Publication: United States NLM ID: 101589536 Publication Model: Print Cited Medium: Internet ISSN: 2168-6157 (Electronic) Linking ISSN: 21686149 NLM ISO Abbreviation: JAMA Neurol Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: <searchLink fieldCode="AU" term="%22Adams+D%22">Adams D</searchLink>; Department of Neurology, Centre Hospitalo Universitaire Bicêtre, Assistance Publique Hopitaux de Paris, Université Paris-Saclay, L'Institut national de la santé et de la recherche médicale U-1195, Le Kremlin Bicêtre Cedex, France.<br /><searchLink fieldCode="AU" term="%22Wixner+J%22">Wixner J</searchLink>; Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden.<br /><searchLink fieldCode="AU" term="%22Polydefkis+M%22">Polydefkis M</searchLink>; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, Maryland.<br /><searchLink fieldCode="AU" term="%22Berk+JL%22">Berk JL</searchLink>; Boston University School of Medicine, Boston, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Conceição+IM%22">Conceição IM</searchLink>; Department of Neurosciences and Mental Health, Unidade Local De Saúde Santa Maria, Hospital de Santa Maria, Centro Académico de Medicina de Lisboa, Lisbon, Portugal.; Instituto de Fisiologia, Instituto de Medicina Molecular, Centro de Estudos Egas Moniz, Faculdade de Medicina, Universidade de Lisboa, Lisbon, Portugal.<br /><searchLink fieldCode="AU" term="%22Dispenzieri+A%22">Dispenzieri A</searchLink>; Division of Hematology, Mayo Clinic, Rochester, Minnesota.<br /><searchLink fieldCode="AU" term="%22Peltier+A%22">Peltier A</searchLink>; Department of Neurology, Vanderbilt University Medical Center, Nashville, Tennessee.<br /><searchLink fieldCode="AU" term="%22Ueda+M%22">Ueda M</searchLink>; Department of Neurology, Kumamoto University Hospital, Kumamoto, Japan.<br /><searchLink fieldCode="AU" term="%22Bender+S%22">Bender S</searchLink>; Alnylam Pharmaceuticals, Cambridge, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Capocelli+K%22">Capocelli K</searchLink>; Alnylam Pharmaceuticals, Cambridge, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Jay+PY%22">Jay PY</searchLink>; Alnylam Pharmaceuticals, Cambridge, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Yureneva+E%22">Yureneva E</searchLink>; Alnylam Pharmaceuticals, Cambridge, Massachusetts.<br /><searchLink fieldCode="AU" term="%22Obici+L%22">Obici L</searchLink>; Amyloidosis Research and Treatment Center, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico Policlinico San Matteo, Pavia, Italy.
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