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TITLE: Design and Rationale of the Global Phase 3 NEURO-TTRansform Study of Antisense Oligonucleotide AKCEA-TTR-L-Rx (ION-682884-CS3) in Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
AUTHORS: Coelho, T; Ando, Y; Benson, MD; Berk, JL; Waddington Cruz, M; Dyck, PJ; Gillmore, JD; Khella, SL; Litchy, WJ; Obici, L; Monteiro, C; Tai, LJ; Viney, NJ; Buchele, G; Brambatti, M; Jung, SW; O'Dea, LS; Tsimikas, S; Schneider, E; Geary, RS; Monia, BP; Gertz, M; ...More
PUBLISHED: 2021, SOURCE: NEUROLOGY AND THERAPY
INDEXED IN: Scopus WOS
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TITLE: Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis  Full Text
AUTHORS: Teresa Coelho; Aaron Yarlas; Marcia Waddington Cruz; Michelle K White; Asia Sikora Kessler; Andrew Lovley; Michael Pollock; Spencer Guthrie; Elizabeth J Ackermann; Steven G Hughes; Chafic Karam; Sami Khella; Morie Gertz; Giampaolo Merlini; Laura Obici; Hartmut H Schmidt; Michael Polydefkis; James J B Dyck; Thomas H Brannagan; Isabel Conceicao; Merrill D Benson; John L Berk; ...More
PUBLISHED: 2020, SOURCE: JOURNAL OF NEUROLOGY, VOLUME: 267, ISSUE: 4
INDEXED IN: Scopus WOS
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TITLE: Plasma neurofilament light chain: an early biomarker for hereditary ATTR amyloid polyneuropathy  Full Text
AUTHORS: Luis F Maia; Aleksandra Maceski; Isabel Conceicao; Laura Obici; Magalhães, R ; Andreas Cortese; David Leppert; Giampaolo Merlini; Jens Kuhle; Maria Joao Saraiva ;
PUBLISHED: 2020, SOURCE: AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, VOLUME: 27, ISSUE: 2
INDEXED IN: Scopus WOS CrossRef: 36
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TITLE: Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis  Full Text
AUTHORS: Laura Obici; John L Berk; Alejandra Gonzalez Duarte; Teresa Coelho; Julian Gillmore; Hartmut H J Schmidt; Matthias Schilling; Taro Yamashita; Celine Labeyrie; Thomas H Brannagan; Senda Ajroud Driss; Peter Gorevic; Arnt V Kristen; Jaclyn Franklin; Jihong H Chen; Marianne T Sweetser; Jing Jing Wang; David Adams;
PUBLISHED: 2020, SOURCE: AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, VOLUME: 27, ISSUE: 3
INDEXED IN: Scopus WOS
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TITLE: A Narrative Review of the Role of Transthyretin in Health and Disease
AUTHORS: Marcia Almeida Liz; Teresa Coelho; Vittorio Bellotti; Maria Isabel Fernandez Arias; Pablo Mallaina; Laura Obici;
PUBLISHED: 2020, SOURCE: NEUROLOGY AND THERAPY
INDEXED IN: Scopus WOS
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TITLE: Transthyretin deposition in the eye in the era of effective therapy for hereditary ATTRV30M amyloidosis  Full Text
AUTHORS: Joel N Buxbaum; Thomas Brannagan; Juan Buades Reines; Eugenia Cisneros; Isabel Conceicao; Theodoros Kyriakides; Giampaolo Merlini; Laura Obici; Violaine Plante Bordeneuve; Antoine Rousseau; Yoshiki Sekijima; Akira Imai; Marcia Waddington Cruz; Masahito Yamada;
PUBLISHED: 2019, SOURCE: AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, VOLUME: 26, ISSUE: 1
INDEXED IN: Scopus WOS
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TITLE: Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis
AUTHORS: Arnt V Kristen; Senda Ajroud Driss; Isabel Conceicao; Peter Gorevic; Theodoros Kyriakides; Laura Obici;
PUBLISHED: 2019, SOURCE: NEURODEGENERATIVE DISEASE MANAGEMENT, VOLUME: 9, ISSUE: 1
INDEXED IN: Scopus WOS
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TITLE: Assessment of patients with hereditary transthyretin amyloidosis–understanding the impact of management and disease progression
AUTHORS: Conceição, I; Coelho, T; Rapezzi, C; Parman, Y; Obici, L; Galán, L; Rousseau, A;
PUBLISHED: 2019, SOURCE: Amyloid, VOLUME: 26, ISSUE: 3
INDEXED IN: Scopus
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TITLE: Assessment of patients with hereditary transthyretin amyloidosis - understanding the impact of management and disease progression  Full Text
AUTHORS: Isabel Conceicao; Teresa Coelho; Claudio Rapezzi; Yesim Parman; Laura Obici; Lucia Galan; Antoine Rousseau;
PUBLISHED: 2019, SOURCE: AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, VOLUME: 26, ISSUE: 3
INDEXED IN: WOS
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TITLE: Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
AUTHORS: Benson, MD; Waddington Cruz, M; Berk, JL; Polydefkis, M; Dyck, PJ; Wang, AK; Plante Bordeneuve, V; Barroso, FA; Merlini, G; Obici, L; Scheinberg, M; Brannagan, TH; Litchy, WJ; Whelan, C; Drachman, BM; Adams, D; Heitner, SB; Conceicao, I; Schmidt, HH; Vita, G; Campistol, JM; Gamez, J; Gorevic, PD; Gane, E; Shah, AM; Solomon, SD; Monia, BP; Hughes, SG; Kwoh, TJ; McEvoy, BW; Jung, SW; Baker, BF; Ackermann, EJ; Gertz, MA; Coelho, T; ...More
PUBLISHED: 2018, SOURCE: NEW ENGLAND JOURNAL OF MEDICINE, VOLUME: 379, ISSUE: 1
INDEXED IN: Scopus WOS
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