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Patient-reported outcomes on familial amyloid polyneuropathy (FAP).
Bolte FJ, Langenstroer C, Friebel F, Hüsing-Kabar A, Dugas M, Schmidt HH. Bolte FJ, et al. Among authors: schmidt hh. Orphanet J Rare Dis. 2020 Oct 14;15(1):287. doi: 10.1186/s13023-020-01575-6. Orphanet J Rare Dis. 2020. PMID: 33054844 Free PMC article.
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.
523 results