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Inotersen preserves or improves quality of life in hereditary transthyretin amyloidosis.
Coelho T, Yarlas A, Waddington-Cruz M, White MK, Sikora Kessler A, Lovley A, Pollock M, Guthrie S, Ackermann EJ, Hughes SG, Karam C, Khella S, Gertz M, Merlini G, Obici L, Schmidt HH, Polydefkis M, Dyck PJB, Brannagan Iii TH, Conceição I, Benson MD, Berk JL. Coelho T, et al. Among authors: schmidt hh. J Neurol. 2020 Apr;267(4):1070-1079. doi: 10.1007/s00415-019-09671-9. Epub 2019 Dec 18. J Neurol. 2020. PMID: 31853709 Free PMC article. Clinical Trial.
[Hereditary amyloidosis].
Schmidt HH, Pröpsting MJ, Manns MP. Schmidt HH, et al. Internist (Berl). 1999 May;40(5):493-501. doi: 10.1007/s001080050363. Internist (Berl). 1999. PMID: 10407759 Review. German. No abstract available.
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. Among authors: schmidt hh. 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.
SERPINA1 modulates expression of amyloidogenic transthyretin.
Niemietz C, Bezerra F, Almeida MR, Guo S, Monia BP, Saraiva MJ, Schütz P, Schmidt HH, Zibert A. Niemietz C, et al. Among authors: schmidt hh. Exp Cell Res. 2020 Oct 15;395(2):112217. doi: 10.1016/j.yexcr.2020.112217. Epub 2020 Aug 5. Exp Cell Res. 2020. PMID: 32768500 Free article.
Analysis of autonomic outcomes in APOLLO, a phase III trial of the RNAi therapeutic patisiran in patients with hereditary transthyretin-mediated amyloidosis.
González-Duarte A, Berk JL, Quan D, Mauermann ML, Schmidt HH, Polydefkis M, Waddington-Cruz M, Ueda M, Conceição IM, Kristen AV, Coelho T, Cauquil CA, Tard C, Merkel M, Aldinc E, Chen J, Sweetser MT, Wang JJ, Adams D. González-Duarte A, et al. Among authors: schmidt hh. J Neurol. 2020 Mar;267(3):703-712. doi: 10.1007/s00415-019-09602-8. Epub 2019 Nov 14. J Neurol. 2020. PMID: 31728713 Free PMC article. Clinical Trial.
520 results