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Page 1
Long-term safety and efficacy of patisiran for hereditary transthyretin-mediated amyloidosis with polyneuropathy: 12-month results of an open-label extension study.
Adams D, Polydefkis M, González-Duarte A, Wixner J, Kristen AV, Schmidt HH, Berk JL, Losada López IA, Dispenzieri A, Quan D, Conceição IM, Slama MS, Gillmore JD, Kyriakides T, Ajroud-Driss S, Waddington-Cruz M, Mezei MM, Planté-Bordeneuve V, Attarian S, Mauricio E, Brannagan TH 3rd, Ueda M, Aldinc E, Wang JJ, White MT, Vest J, Berber E, Sweetser MT, Coelho T; patisiran Global OLE study group. Adams D, et al. Lancet Neurol. 2021 Jan;20(1):49-59. doi: 10.1016/S1474-4422(20)30368-9. Epub 2020 Nov 16. Lancet Neurol. 2021. PMID: 33212063 Clinical Trial.
Recommendations for the diagnosis and management of transthyretin amyloidosis with gastrointestinal manifestations.
Nakov R, Suhr OB, Ianiro G, Kupcinskas J, Segal JP, Dumitrascu DL, Heinrich H, Mikolasevic I, Stojkovic-Lalosevic M, Barbov I, Sarafov S, Tournev I, Nakov V, Wixner J. Nakov R, et al. Among authors: sarafov s. Eur J Gastroenterol Hepatol. 2021 May 1;33(5):613-622. doi: 10.1097/MEG.0000000000002030. Eur J Gastroenterol Hepatol. 2021. PMID: 33394808 Review.
Clinical trials in pediatric ALS: a TRICALS feasibility study.
Kliest T, Van Eijk RPA, Al-Chalabi A, Albanese A, Andersen PM, Amador MDM, BrÅthen G, Brunaud-Danel V, Brylev L, Camu W, De Carvalho M, Cereda C, Cetin H, Chaverri D, Chiò A, Corcia P, Couratier P, De Marchi F, Desnuelle C, Van Es MA, Esteban J, Filosto M, GarcÍa Redondo A, Grosskreutz J, Hanemann CO, HolmØy T, HØyer H, Ingre C, Koritnik B, Kuzma-Kozakiewicz M, Lambert T, Leigh PN, Lunetta C, Mandrioli J, Mcdermott CJ, Meyer T, Mora JS, Petri S, Povedano M, Reviers E, Riva N, Roes KCB, Rubio MÁ, Salachas F, Sarafov S, SorarÙ G, Stevic Z, Svenstrup K, MØller AT, Turner MR, Van Damme P, Van Leeuwen LAG, Varona L, VÁzquez Costa JF, Weber M, Hardiman O, Van Den Berg LH. Kliest T, et al. Among authors: sarafov s. Amyotroph Lateral Scler Frontotemporal Degener. 2022 Nov;23(7-8):481-488. doi: 10.1080/21678421.2021.2024856. Epub 2022 Feb 16. Amyotroph Lateral Scler Frontotemporal Degener. 2022. PMID: 35172656 Free PMC article.
Founder effect of the Glu89Gln TTR mutation in the Bulgarian population.
Kirov A, Sarafov S, Pavlova Z, Todorov T, Chamova T, Gospodinova M, Tournev I, Mitev V, Todorova A. Kirov A, et al. Among authors: sarafov s. Amyloid. 2019 Dec;26(4):181-185. doi: 10.1080/13506129.2019.1634539. Epub 2019 Jul 29. Amyloid. 2019. PMID: 31353960
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