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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.
Adams, David; Polydefkis, Michael; González-Duarte, Alejandra; Wixner, Jonas; Kristen, Arnt V; Schmidt, Hartmut H; Berk, John L; Losada López, Inés Asunción; Dispenzieri, Angela; Quan, Dianna; Conceição, Isabel M; Slama, Michel S; Gillmore, Julian D; Kyriakides, Theodoros; Ajroud-Driss, Senda; Waddington-Cruz, Márcia; Mezei, Michelle M; Planté-Bordeneuve, Violaine; Attarian, Shahram; Mauricio, Elizabeth; Brannagan, Thomas H; Ueda, Mitsuharu; Aldinc, Emre; Wang, Jing Jing; White, Matthew T; Vest, John; Berber, Erhan; Sweetser, Marianne T; Coelho, Teresa.
Afiliação
  • Adams D; Université Paris-Saclay, U1195, INSERM, Le Kremlin Bicêtre, France; Neurology Department, AP-HP, Centre Hospitalier Universitaire Bicêtre, Le Kremlin Bicêtre, France. Electronic address: david.adams@aphp.fr.
  • Polydefkis M; Department of Neurology, Johns Hopkins University School of Medicine, Baltimore, MD, USA.
  • González-Duarte A; Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Mexico City, Mexico.
  • Wixner J; Department of Public Health and Clinical Medicine, Umeå University, Umeå, Sweden.
  • Kristen AV; Department of Cardiology, University of Heidelberg, Heidelberg, Germany.
  • Schmidt HH; University of Münster, Münster, Germany.
  • Berk JL; Boston Medical Center, Boston, MA, USA.
  • Losada López IA; Balearic Islands Health Research Institute, Palma, Spain.
  • Dispenzieri A; Division of Hematology, Mayo Clinic, Rochester, MN, USA.
  • Quan D; University of Colorado, Denver, CO, USA.
  • Conceição IM; Centro Hospitalar Universitário Lisboa Norte, Hospital de Santa Maria and Faculdade de Medicina, Lisbon, Portugal.
  • Slama MS; Cardiology Department, Centre de Compétence Amylose Cardiaque, Centre Hospitalier Universitaire Xavier Bichat Assistance Publique Hôpitaux de Paris, Université Paris-Saclay, Paris, France.
  • Gillmore JD; National Amyloidosis Centre, University College London, Royal Free Hospital, London, UK.
  • Kyriakides T; The Cyprus Institute of Neurology and Genetics, Nicosia, Cyprus; Medical School, University of Nicosia, Nicosia, Cyprus.
  • Ajroud-Driss S; Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL, USA.
  • Waddington-Cruz M; Hospital Universitario Clementino Fraga Filho-Universidade Federal do Rio de Janeiro, Rio de Janeiro, Brazil.
  • Mezei MM; Division of Neurology, Department of Medicine, University of British Columbia, Vancouver, BC, Canada.
  • Planté-Bordeneuve V; Centre Hospitalier Universitaire, Henri Mondor-Assistance Publique Hopitaux de Paris, Univ Paris Est Creteil, INSERM, IMRB, Creteil, France.
  • Attarian S; Neuromuscular disorders and ALS Department, Centre Hospitalier Universitaire La Timone, Marseille, France.
  • Mauricio E; Mayo Clinic, Jacksonville, FL, USA.
  • Brannagan TH; Columbia University, College of Physicians and Surgeons, Neurology Department, New York, NY, USA.
  • Ueda M; Kumamoto University Hospital, Department of Neurology, Kumamoto, Japan.
  • Aldinc E; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Wang JJ; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • White MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Vest J; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Berber E; Alnylam Pharmaceuticals, Cambridge, MA, USA; Myokardia, Brisbane, CA, USA.
  • Sweetser MT; Alnylam Pharmaceuticals, Cambridge, MA, USA.
  • Coelho T; Centro Hospitalar Universitário do Porto, Porto, Portugal.
Lancet Neurol ; 20(1): 49-59, 2021 01.
Article em En | MEDLINE | ID: mdl-33212063
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Polineuropatias / Pré-Albumina / Avaliação de Resultados em Cuidados de Saúde / Neuropatias Amiloides Familiares / RNA Interferente Pequeno / Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos Tipo de estudo: Clinical_trials / Etiology_studies Limite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Polineuropatias / Pré-Albumina / Avaliação de Resultados em Cuidados de Saúde / Neuropatias Amiloides Familiares / RNA Interferente Pequeno / Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos Tipo de estudo: Clinical_trials / Etiology_studies Limite: Adult / Aged / Female / Humans / Male / Middle aged Idioma: En Ano de publicação: 2021 Tipo de documento: Article