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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. 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.
Familial dysautonomia.
González-Duarte A, Cotrina-Vidal M, Kaufmann H, Norcliffe-Kaufmann L. González-Duarte A, et al. Clin Auton Res. 2023 Jun;33(3):269-280. doi: 10.1007/s10286-023-00941-1. Epub 2023 May 19. Clin Auton Res. 2023. PMID: 37204536 Review.
Familial amyloidosis with polyneuropathy associated with TTR Ser50Arg mutation.
González-Duarte A, Soto KC, Martínez-Baños D, Arteaga-Vazquez J, Barrera F, Berenguer-Sanchez M, Cantu-Brito C, García-Ramos G, Estañol Vidal B. González-Duarte A, et al. Amyloid. 2012 Dec;19(4):171-6. doi: 10.3109/13506129.2012.712925. Epub 2012 Aug 28. Amyloid. 2012. PMID: 22928869
Effects of Patisiran, an RNA Interference Therapeutic, on Cardiac Parameters in Patients With Hereditary Transthyretin-Mediated Amyloidosis.
Solomon SD, Adams D, Kristen A, Grogan M, González-Duarte A, Maurer MS, Merlini G, Damy T, Slama MS, Brannagan TH 3rd, Dispenzieri A, Berk JL, Shah AM, Garg P, Vaishnaw A, Karsten V, Chen J, Gollob J, Vest J, Suhr O. Solomon SD, et al. Circulation. 2019 Jan 22;139(4):431-443. doi: 10.1161/CIRCULATIONAHA.118.035831. Circulation. 2019. PMID: 30586695 Clinical Trial.
92 results