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Efficacy and safety of vutrisiran for patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy: a randomized clinical trial.
Adams D, Tournev IL, Taylor MS, Coelho T, Planté-Bordeneuve V, Berk JL, González-Duarte A, Gillmore JD, Low SC, Sekijima Y, Obici L, Chen C, Badri P, Arum SM, Vest J, Polydefkis M; HELIOS-A Collaborators. Adams D, et al. Amyloid. 2023 Mar;30(1):1-9. doi: 10.1080/13506129.2022.2091985. Epub 2022 Jul 23. Amyloid. 2023. PMID: 35875890 Free article. Clinical Trial.
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: langenstroer c. 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.