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. |
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| 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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| ISSN: | 2168-6157 |
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| DOI: | 10.1001/jamaneurol.2024.4631 |
