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Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis.
Benson MD, Waddington-Cruz M, Berk JL, Polydefkis M, Dyck PJ, Wang AK, Planté-Bordeneuve V, Barroso FA, Merlini G, Obici L, Scheinberg M, Brannagan TH 3rd, Litchy WJ, Whelan C, Drachman BM, Adams D, Heitner SB, Conceição 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. Benson MD, et al. N Engl J Med. 2018 Jul 5;379(1):22-31. doi: 10.1056/NEJMoa1716793. N Engl J Med. 2018. PMID: 29972757 Free article. Clinical Trial.
Familial amyloid polyneuropathy.
Planté-Bordeneuve V, Said G. Planté-Bordeneuve V, et al. Lancet Neurol. 2011 Dec;10(12):1086-97. doi: 10.1016/S1474-4422(11)70246-0. Lancet Neurol. 2011. PMID: 22094129 Review.
Hereditary systemic amyloidosis due to Asp76Asn variant β2-microglobulin.
Valleix S, Gillmore JD, Bridoux F, Mangione PP, Dogan A, Nedelec B, Boimard M, Touchard G, Goujon JM, Lacombe C, Lozeron P, Adams D, Lacroix C, Maisonobe T, Planté-Bordeneuve V, Vrana JA, Theis JD, Giorgetti S, Porcari R, Ricagno S, Bolognesi M, Stoppini M, Delpech M, Pepys MB, Hawkins PN, Bellotti V. Valleix S, et al. N Engl J Med. 2012 Jun 14;366(24):2276-83. doi: 10.1056/NEJMoa1201356. N Engl J Med. 2012. PMID: 22693999 Free article.
Tafamidis for transthyretin familial amyloid polyneuropathy: a randomized, controlled trial.
Coelho T, Maia LF, Martins da Silva A, Waddington Cruz M, Planté-Bordeneuve V, Lozeron P, Suhr OB, Campistol JM, Conceição IM, Schmidt HH, Trigo P, Kelly JW, Labaudinière R, Chan J, Packman J, Wilson A, Grogan DR. Coelho T, et al. Neurology. 2012 Aug 21;79(8):785-92. doi: 10.1212/WNL.0b013e3182661eb1. Epub 2012 Jul 25. Neurology. 2012. PMID: 22843282 Free PMC article. Clinical Trial.
Guideline of transthyretin-related hereditary amyloidosis for clinicians.
Ando Y, Coelho T, Berk JL, Cruz MW, Ericzon BG, Ikeda S, Lewis WD, Obici L, Planté-Bordeneuve V, Rapezzi C, Said G, Salvi F. Ando Y, et al. Orphanet J Rare Dis. 2013 Feb 20;8:31. doi: 10.1186/1750-1172-8-31. Orphanet J Rare Dis. 2013. PMID: 23425518 Free PMC article. Review.
Transthyretin familial amyloid polyneuropathy.
Planté-Bordeneuve V, Kerschen P. Planté-Bordeneuve V, et al. Handb Clin Neurol. 2013;115:643-58. doi: 10.1016/B978-0-444-52902-2.00038-2. Handb Clin Neurol. 2013. PMID: 23931808 Review.
125 results