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Cardiac amyloidosis: An update on pathophysiology, diagnosis, and treatmentSiddiqi, Omar K. ; Ruberg, Frederick L.Trends in cardiovascular medicine, 2018-01, Vol.28 (1), p.10-21 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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Genotype and Phenotype of Transthyretin Cardiac Amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey)Maurer, Mathew S ; Hanna, Mazen ; Grogan, Martha ; Dispenzieri, Angela ; Witteles, Ronald ; Drachman, Brian ; Judge, Daniel P ; Lenihan, Daniel J ; Gottlieb, Stephen S ; Shah, Sanjiv J ; Steidley, D Eric ; Ventura, Hector ; Murali, Srinivas ; Silver, Marc A ; Jacoby, Daniel ; Fedson, Savitri ; Hummel, Scott L ; Kristen, Arnt V ; Damy, Thibaud ; Planté-Bordeneuve, Violaine ; Coelho, Teresa ; Mundayat, Rajiv ; Suhr, Ole B ; Waddington Cruz, Márcia ; Rapezzi, ClaudioJournal of the American College of Cardiology, 2016-07, Vol.68 (2), p.161-172 [Periódico revisado por pares]United States: Elsevier LimitedTexto completo disponível |
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Material Type: Artigo
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Lipid Nanoparticle Systems for Enabling Gene TherapiesCullis, Pieter R. ; Hope, Michael J.Molecular therapy, 2017-07, Vol.25 (7), p.1467-1475 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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Material Type: Artigo
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RNA-targeting and gene editing therapies for transthyretin amyloidosisAimo, Alberto ; Castiglione, Vincenzo ; Rapezzi, Claudio ; Franzini, Maria ; Panichella, Giorgia ; Vergaro, Giuseppe ; Gillmore, Julian ; Fontana, Marianna ; Passino, Claudio ; Emdin, MicheleNature reviews cardiology, 2022-10, Vol.19 (10), p.655-667 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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Material Type: Artigo
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Hereditary transthyretin amyloidosis: a model of medical progress for a fatal diseaseAdams, David ; Koike, Haruki ; Slama, Michel ; Coelho, TeresaNature reviews. Neurology, 2019-07, Vol.15 (7), p.387-404 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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Material Type: Artigo
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CRISPR-Cas9 In Vivo Gene Editing for Transthyretin AmyloidosisGillmore, Julian D ; Gane, Ed ; Taubel, Jorg ; Kao, Justin ; Fontana, Marianna ; Maitland, Michael L ; Seitzer, Jessica ; O’Connell, Daniel ; Walsh, Kathryn R ; Wood, Kristy ; Phillips, Jonathan ; Xu, Yuanxin ; Amaral, Adam ; Boyd, Adam P ; Cehelsky, Jeffrey E ; McKee, Mark D ; Schiermeier, Andrew ; Harari, Olivier ; Murphy, Andrew ; Kyratsous, Christos A ; Zambrowicz, Brian ; Soltys, Randy ; Gutstein, David E ; Leonard, John ; Sepp-Lorenzino, Laura ; Lebwohl, DavidThe New England journal of medicine, 2021-08, Vol.385 (6), p.493-502 [Periódico revisado por pares]United States: Massachusetts Medical SocietyTexto completo disponível |
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Material Type: Artigo
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Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatmentsSekijima, YoshikiJournal of neurology, neurosurgery and psychiatry, 2015-09, Vol.86 (9), p.1036-1043 [Periódico revisado por pares]EnglandTexto completo disponível |
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Material Type: Artigo
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Transthyretin Amyloid Cardiomyopathy: JACC State-of-the-Art ReviewRuberg, Frederick L ; Grogan, Martha ; Hanna, Mazen ; Kelly, Jeffery W ; Maurer, Mathew SJournal of the American College of Cardiology, 2019-06, Vol.73 (22), p.2872-2891 [Periódico revisado por pares]United States: Elsevier LimitedTexto completo disponível |
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Material Type: Artigo
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Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trialAdams, David ; Tournev, Ivailo L. ; Taylor, Mark S. ; Coelho, Teresa ; Planté-Bordeneuve, Violaine ; Berk, John L. ; González-Duarte, Alejandra ; Gillmore, Julian D. ; Low, Soon-Chai ; Sekijima, Yoshiki ; Obici, Laura ; Chen, Chongshu ; Badri, Prajakta ; Arum, Seth M. ; Vest, John ; Polydefkis, MichaelAmyloid, 2023-01, Vol.30 (1), p.18-26 [Periódico revisado por pares]England: Taylor & FrancisTexto completo disponível |
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Material Type: Artigo
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Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathyCoelho, Teresa ; Maia, Luis F. ; da Silva, Ana Martins ; Cruz, Márcia W. ; Planté-Bordeneuve, Violaine ; Suhr, Ole B. ; Conceiçao, Isabel ; Schmidt, Hartmut H.-J. ; Trigo, Pedro ; Kelly, Jeffery W. ; Labaudinière, Richard ; Chan, Jason ; Packman, Jeff ; Grogan, Donna R.Journal of neurology, 2013-11, Vol.260 (11), p.2802-2814 [Periódico revisado por pares]Berlin/Heidelberg: Springer Berlin HeidelbergTexto completo disponível |