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Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study

David Adams, Michael Polydefkis, Alejandra González‐Duarte, Jonas Wixner, Arnt V. Kristen, Hartmut Schmidt, John L. Berk, Inés Asunción Losada López, Angela Dispenzieri, Dianna Quan, Isabel Conceição, Michel Slama, Julian D. Gillmore, Theodoros Kyriakides, Senda Ajroud‐Driss, Márcia Waddington‐Cruz, Michelle M. Mezei, Violaine Planté‐Bordeneuve, Shahram Attarian, Elizabeth A. Mauricio, Thomas H. Brannagan, Mitsuharu Ueda, Emre Aldinc, Jing Jing Wang, Matthew T. White, John Vest, Erhan Berber, Marianne T. Sweetser, Teresa Coelho, Giuseppe Vita, Vincenzo Rizzo, Massimo Russo, Anna Mazzeo, Luca Gentile, John L. Berk, Caitlin Brueckner, Victoria Lazzari, Janice F. Wiesman, Douglas DeLong, Jennifer Victory, James A. R. Dalton, John J. May, Catherine Gilmore, Shahram Attarian, Saran Diallo, Émilien Delmont, Jean Pouget, Annie Verschueren, Aude‐Marie Grapperon, Emmanuelle Salort‐Campana, Isabel Conceição, Ana Tarina Alvarez Lopes, Filipa Lamas, Carlos Marques‐Neves, José Castro, Pedro Pereira, Isabel Castro, Ana Franco, Miguel Oliveira Santos, Conceição de Azevedo Coutinho, Catarina Falcão de Campos, Teresa Coelho, Antonio Hipólito Reis, Nuno Correia, Javier Martínez Pérez, Ana Silva, Cristina Alves, Márcio Cardoso, Kátia Valdrez, Julia R Monte, Bernardete Pessoa, Nádia Guimarães, Mónica Freitas, Joana Ramalho, Natália Ferreira, Daisuke Kuzume, Céline Tard, Nawal Waucquier, Isabelle Rougeaux, Sylvie Brice, Emmanuelle Kasprzyk, Elise Elrezzi, Sayah Meguig, É. Hachulla, Clément Gauvain, Maria-Claire Migaud-Chervy, Dominique Deplanque, Elsa Jozefowicz, Loïc Lebellec, David B. Adams, Line Balaya-Gouraya, Nathalie Jehan Lacour, Halima Bournane, Nathalie Martin, Mongia Elabed, Niamey Sacko, Yasmine Boubrit, Amina Gaouar, Fetra Rakotondratafika, Marie Théaudin-Saliou

2020The Lancet Neurology156 citationsDOI

Topics & Concepts

MedicineTransthyretinPolyneuropathyClinical trialAmyloidosisInternal medicinePlaceboPediatricsPhysical therapyPathologyAlternative medicineAmyloidosis: Diagnosis, Treatment, OutcomesSkin and Cellular Biology ResearchAlzheimer's disease research and treatments
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study | Litcius