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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
12
TITLE: Impact of Patisiran, an investigational RNAi Therapeutic, on nutritional status in patients with hereditary Transthyretin-Mediated Amyloidosis  Full Text
AUTHORS: Obici, L; Coelho, T; Adams, D; Gonzalez Duarte, A; O'Riordan, W; Yang, CC; Yamashita, T; Kristen, A; Tournev, I; Schmidt, H; Berk, J; Lin, KP; Gandhi, P; Sweetser, M; Chen, J; Goyal, S; Gollob, J; Suhr, O;
PUBLISHED: 2018, SOURCE: 4th Congress of the European-Academy-of-Neurology (EAN) in EUROPEAN JOURNAL OF NEUROLOGY, VOLUME: 25
INDEXED IN: WOS
13
TITLE: "Red-flag" symptom clusters in transthyretin familial amyloid polyneuropathy  Full Text
AUTHORS: Isabel Conceicao; Alejandra Gonzalez Duarte; Laura Obici; Hartmut H J Schmidt; Damien Simoneau; Moh Lim Ong; Leslie Amass;
PUBLISHED: 2016, SOURCE: JOURNAL OF THE PERIPHERAL NERVOUS SYSTEM, VOLUME: 21, ISSUE: 1
INDEXED IN: WOS
14
TITLE: Management of asymptomatic gene carriers of transthyretin familial amyloid polyneuropathy. TTR-FAP Gene Carriers  Full Text
AUTHORS: Hartmut H J Schmidt; Fabio Barroso; Alejandra Gonzalez Duarte; Isabel Conceicao; Laura Obici; Denis Keohane; Leslie Amass;
PUBLISHED: 2016, SOURCE: MUSCLE & NERVE, VOLUME: 54, ISSUE: 3
INDEXED IN: Scopus WOS CrossRef
15
TITLE: Recommendations for presymptomatic genetic testing and management of individuals at risk for hereditary transthyretin amyloidosis
AUTHORS: Laura Obici; Jan B Kuks; Juan Buades; David Adams; Ole B Suhr; Teresa Coelho; Theodore Kyriakides;
PUBLISHED: 2016, SOURCE: CURRENT OPINION IN NEUROLOGY, VOLUME: 29
INDEXED IN: WOS
16
TITLE: Rapid progression of familial amyloidotic polyneuropathy A multinational natural history study
AUTHORS: David Adams; Teresa Coelho; Laura Obici; Giampaolo Merlini; Zoia Mincheva; Narupat Suanprasert; Brian R Bettencourt; Jared A Gollob; Pritesh J Gandhi; William J Litchy; Peter J Dyck;
PUBLISHED: 2015, SOURCE: NEUROLOGY, VOLUME: 85, ISSUE: 8
INDEXED IN: Scopus WOS CrossRef
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TITLE: Guideline of transthyretin-related hereditary amyloidosis for clinicians  Full Text
AUTHORS: Yukio Ando; Teresa Coelho; John L Berk; Marcia Waddington Cruz; Bo Goran Ericzon; Shu ichi Ikeda; David D Lewis; Laura Obici; Violaine Plante Bordeneuve; Claudio Rapezzi; Gerard Said; Fabrizio Salvi;
PUBLISHED: 2013, SOURCE: ORPHANET JOURNAL OF RARE DISEASES, VOLUME: 8, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
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TITLE: Doxycycline plus tauroursodeoxycholic acid for transthyretin amyloidosis: a phase II study  Full Text
AUTHORS: Laura Obici; Andrea Cortese; Alessandro Lozza; Lucchetti, J; Marco Gobbi; Giovanni Palladini; Stefano Perlini; Maria J Saraiva ; Giampaolo Merlini;
PUBLISHED: 2012, SOURCE: AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS, VOLUME: 19, ISSUE: SUPPL. 1
INDEXED IN: Scopus WOS CrossRef: 153
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