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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.
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.
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.
Five-Year Results With Patisiran for Hereditary Transthyretin Amyloidosis With Polyneuropathy: A Randomized Clinical Trial With Open-Label Extension.
Adams D, Wixner J, Polydefkis M, Berk JL, Conceição IM, Dispenzieri A, Peltier A, Ueda M, Bender S, Capocelli K, Jay PY, Yureneva E, Obici L; patisiran Global OLE study group. Adams D, et al. JAMA Neurol. 2025 Jan 13. doi: 10.1001/jamaneurol.2024.4631. Online ahead of print. JAMA Neurol. 2025. PMID: 39804640
62 results