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Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis.
Adams D, Gonzalez-Duarte A, O'Riordan WD, Yang CC, Ueda M, Kristen AV, Tournev I, Schmidt HH, Coelho T, Berk JL, Lin KP, Vita G, Attarian S, Planté-Bordeneuve V, Mezei MM, Campistol JM, Buades J, Brannagan TH 3rd, Kim BJ, Oh J, Parman Y, Sekijima Y, Hawkins PN, Solomon SD, Polydefkis M, Dyck PJ, Gandhi PJ, Goyal S, Chen J, Strahs AL, Nochur SV, Sweetser MT, Garg PP, Vaishnaw AK, Gollob JA, Suhr OB. Adams D, et al. Among authors: sekijima y. N Engl J Med. 2018 Jul 5;379(1):11-21. doi: 10.1056/NEJMoa1716153. N Engl J Med. 2018. PMID: 29972753 Free article. Clinical Trial.
The diflunisal trial: update on study drug tolerance and disease progression.
Berk JL, Dyck PJ, Obici L, Zeldenrust SR, Sekijima Y, Yamashita T, Ando Y, Ikeda SI, Gorevic P, Merlini G, Kelly JW, Skinner M, Bisbee AB, Suhr OB; Familial Amyloidosis Consortium. Berk JL, et al. Among authors: sekijima y. Amyloid. 2011 Jun;18 Suppl 1:196-7. doi: 10.3109/13506129.2011.574354073. Amyloid. 2011. PMID: 21838485 No abstract available.
The Diflunisal Trial: study accrual and drug tolerance.
Berk JL, Suhr OB, Sekijima Y, Yamashita T, Heneghan M, Zeldenrust SR, Ando Y, Ikeda S, Gorevic P, Merlini G, Kelly JW, Skinner M, Bisbee AB, Dyck PJ, Obici L; Familial Amyloidosis Consortium. Berk JL, et al. Among authors: sekijima y. Amyloid. 2012 Jun;19 Suppl 1:37-8. doi: 10.3109/13506129.2012.678509. Epub 2012 May 2. Amyloid. 2012. PMID: 22551208 Clinical Trial.
Repurposing diflunisal for familial amyloid polyneuropathy: a randomized clinical trial.
Berk JL, Suhr OB, Obici L, Sekijima Y, Zeldenrust SR, Yamashita T, Heneghan MA, Gorevic PD, Litchy WJ, Wiesman JF, Nordh E, Corato M, Lozza A, Cortese A, Robinson-Papp J, Colton T, Rybin DV, Bisbee AB, Ando Y, Ikeda S, Seldin DC, Merlini G, Skinner M, Kelly JW, Dyck PJ; Diflunisal Trial Consortium. Berk JL, et al. Among authors: sekijima y. JAMA. 2013 Dec 25;310(24):2658-67. doi: 10.1001/jama.2013.283815. JAMA. 2013. PMID: 24368466 Free PMC article. Clinical Trial.
Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met: A phase III, open-label study.
Ando Y, Sekijima Y, Obayashi K, Yamashita T, Ueda M, Misumi Y, Morita H, Machii K, Ohta M, Takata A, Ikeda S. Ando Y, et al. Among authors: sekijima y. J Neurol Sci. 2016 Mar 15;362:266-71. doi: 10.1016/j.jns.2016.01.046. Epub 2016 Jan 22. J Neurol Sci. 2016. PMID: 26944161 Clinical Trial.
299 results