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TÍTULO: 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
AUTORES: 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; ...Mais
PUBLICAÇÃO: 2021, FONTE: NEUROLOGY AND THERAPY
INDEXADO EM: Scopus WOS
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TÍTULO: ATTR amyloidosis during the COVID-19 pandemic: insights from a global medical roundtable  Full Text
AUTORES: Brannagan, TH; Auer Grumbach, M; Berk, JL; Briani, C; Bril, V; Coelho, T; Damy, T; Dispenzieri, A; Drachman, BM; Fine, N; Gaggin, HK; Gertz, M; Gillmore, JD; Gonzalez, E; Hanna, M; Hurwitz, DR; Khella, SL; Maurer, MS; Nativi Nicolau, J; Olugemo, K; Quintana, LF; Rosen, AM; Schmidt, HH; Shehata, J; Waddington Cruz, M; Whelan, C; Ruberg, FL; ...Mais
PUBLICAÇÃO: 2021, FONTE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 16, NÚMERO: 1
INDEXADO EM: Scopus WOS
13
TÍTULO: HELIOS-A: 9-month results from the phase 3 study of vutrisiran in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy  Full Text
AUTORES: Adams, D; Tournev, I; Taylor, M; Coelho, T; Plante Bordeneuve, V; Berk, J; Gonzalez Duarte, A; Gillmore, J; Low, SC; Sekijima, Y; Obici, L; Blakesley, R; Arum, S; Shilling, R; Vest, J; Polydefkis, M;
PUBLICAÇÃO: 2021, FONTE: JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, VOLUME: 26, NÚMERO: 3
INDEXADO EM: WOS
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TÍTULO: Efficacy and safety with > 3 years of inotersen treatment for hereditary transthyretin amyloid polyneuropathy  Full Text
AUTORES: Brannagan, T; Wang, A; Polydefkis, M; Dyck, P; Berk, J; Drachman, B; Gorevic, P; Coelho, T; Whelan, C; Conceicao, I; Plante Bordeneuve, V; Merlini, G; Obici, L; Schmidt, H; Plana, JMC; Gamez, J; Kristen, A; Mazzeo, A; Gentile, L; Narayana, A; Olugemo, K; Aquino, P; Benson, M; Gertz, M; ...Mais
PUBLICAÇÃO: 2021, FONTE: JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, VOLUME: 26, NÚMERO: 3
INDEXADO EM: WOS
15
TÍTULO: HELIOS-A: 9-month Results from the Phase 3 Study of Vutrisiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy
AUTORES: Adams, D; Tournev, IL; Taylor, MS; Coelho, T; Plante Bordeneuve, V; Berk, JL; Gonzalez Duarte, A; Gillmore, JD; Low, SC; Sekijima, Y; Obici, L; Blakesley, R; Arum, S; Shilling, R; Vest, J; Polydefkis, M;
PUBLICAÇÃO: 2021, FONTE: NEUROLOGY, VOLUME: 96, NÚMERO: 15
INDEXADO EM: WOS
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TÍTULO: Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis  Full Text
AUTORES: Alejandra Gonzalez Duarte; John L Berk; Dianna N Quan; Michelle L Mauermann; Hartmut H Schmidt; Michael Polydefkis; Marcia Waddington Cruz; Mitsuharu Ueda; Isabel M Conceicao; Arnt V Kristen; Teresa Coelho; Cecile A Cauquil; Celine Tard; Madeline Merkel; Emre Aldinc; Jihong H Chen; Marianne T Sweetser; Jing Jing Wang; David Adams;
PUBLICAÇÃO: 2020, FONTE: JOURNAL OF NEUROLOGY, VOLUME: 267, NÚMERO: 3
INDEXADO EM: Scopus WOS
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TÍTULO: Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis  Full Text
AUTORES: 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; ...Mais
PUBLICAÇÃO: 2020, FONTE: JOURNAL OF NEUROLOGY, VOLUME: 267, NÚMERO: 4
INDEXADO EM: Scopus WOS
18
TÍTULO: Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis (vol 264, pg 703, 2020)  Full Text
AUTORES: Alejandra Gonzalez Duarte; John L Berk; Dianna N Quan; Michelle L Mauermann; Hartmut H Schmidt; Michael Polydefkis; Marcia Waddington Cruz; Mitsuharu Ueda; Isabel M Conceicao; Arnt V Kristen; Teresa Coelho; Cecile A Cauquil; Celine Tard; Madeline Merkel; Emre Aldinc; Jihong H Chen; Marianne T Sweetser; Jing Jing Wang; David Adams;
PUBLICAÇÃO: 2020, FONTE: JOURNAL OF NEUROLOGY, VOLUME: 267, NÚMERO: 3
INDEXADO EM: WOS
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TÍTULO: Quality of life outcomes in APOLLO, the phase 3 trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis  Full Text
AUTORES: 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;
PUBLICAÇÃO: 2020, FONTE: AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, VOLUME: 27, NÚMERO: 3
INDEXADO EM: Scopus WOS
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TÍTULO: A phase II, open-label, extension study of long-term patisiran treatment in patients with hereditary transthyretin-mediated (hATTR) amyloidosis  Full Text
AUTORES: Teresa Coelho; David Adams; Isabel Conceicao; Marcia Waddington Cruz; Hartmut H Schmidt; Juan Buades; Josep Campistol; John L Berk; Michael Polydefkis; Jing Jing Wang; Jihong H Chen; Marianne T Sweetser; Jared Gollob; Ole B Suhr;
PUBLICAÇÃO: 2020, FONTE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 15, NÚMERO: 1
INDEXADO EM: Scopus WOS
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