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Peptide probes detect misfolded transthyretin oligomers in plasma of hereditary amyloidosis patients.
Schonhoft JD, Monteiro C, Plate L, Eisele YS, Kelly JM, Boland D, Parker CG, Cravatt BF, Teruya S, Helmke S, Maurer M, Berk J, Sekijima Y, Novais M, Coelho T, Powers ET, Kelly JW. Schonhoft JD, et al. Among authors: novais m. Sci Transl Med. 2017 Sep 13;9(407):eaam7621. doi: 10.1126/scitranslmed.aam7621. Sci Transl Med. 2017. PMID: 28904227 Free PMC article.
Predictive model of response to tafamidis in hereditary ATTR polyneuropathy.
Monteiro C, Mesgazardeh JS, Anselmo J, Fernandes J, Novais M, Rodrigues C, Brighty GJ, Powers DL, Powers ET, Coelho T, Kelly JW. Monteiro C, et al. Among authors: novais m. JCI Insight. 2019 Jun 20;4(12):e126526. doi: 10.1172/jci.insight.126526. eCollection 2019 Jun 20. JCI Insight. 2019. PMID: 31217346 Free PMC article.
Patisiran treatment in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy after liver transplantation.
Schmidt HH, Wixner J, Planté-Bordeneuve V, Muñoz-Beamud F, Lladó L, Gillmore JD, Mazzeo A, Li X, Arum S, Jay PY, Adams D; Patisiran Post-LT Study Group. Schmidt HH, et al. Am J Transplant. 2022 Jun;22(6):1646-1657. doi: 10.1111/ajt.17009. Epub 2022 Mar 26. Am J Transplant. 2022. PMID: 35213769 Free PMC article. Clinical Trial.
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.
Tafamidis polyneuropathy amelioration requires modest increases in transthyretin stability even though increases in plasma native TTR and decreases in non-native TTR do not predict response.
Monteiro C, Mesgarzadeh JS, Anselmo J, Fernandes J, Novais M, Rodrigues C, Powers DL, Powers ET, Coelho T, Kelly JW. Monteiro C, et al. Among authors: novais m. Amyloid. 2023 Mar;30(1):81-95. doi: 10.1080/13506129.2022.2126308. Epub 2022 Sep 30. Amyloid. 2023. PMID: 36178172 Free PMC article.
Impact of Vutrisiran on Quality of Life and Physical Function in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy.
Obici L, Ajroud-Driss S, Lin KP, Berk JL, Gillmore JD, Kale P, Koike H, Danese D, Aldinc E, Chen C, Vest J, Adams D; HELIOS-A Collaborators Study Group. Obici L, et al. Neurol Ther. 2023 Oct;12(5):1759-1775. doi: 10.1007/s40120-023-00522-4. Epub 2023 Jul 31. Neurol Ther. 2023. PMID: 37523143 Free PMC article.
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