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Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid.
Garcia-Pavia P, Aus dem Siepen F, Donal E, Lairez O, van der Meer P, Kristen AV, Mercuri MF, Michalon A, Frost RJA, Grimm J, Nitsch RM, Hock C, Kahr PC, Damy T. Garcia-Pavia P, et al. Among authors: kristen av. N Engl J Med. 2023 Jul 20;389(3):239-250. doi: 10.1056/NEJMoa2303765. Epub 2023 May 20. N Engl J Med. 2023. PMID: 37212440 Clinical Trial.
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: kristen av. 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.
Patients with transthyretin amyloidosis enrolled in THAOS between 2018 and 2021 continue to experience substantial diagnostic delay.
Coelho T, Dispenzieri A, Grogan M, Conceição I, Waddington-Cruz M, Kristen AV, Wixner J, Diemberger I, Gonzalez-Moreno J, Maurer MS, Planté-Bordeneuve V, Garcia-Pavia P, Tournev I, Gonzalez-Costello J, Cariou E, González-Duarte A, Glass O, Chapman D, Amass L; THAOS investigators. Coelho T, et al. Among authors: kristen av. Amyloid. 2023 Dec;30(4):445-448. doi: 10.1080/13506129.2023.2229484. Epub 2023 Jul 17. Amyloid. 2023. PMID: 37459334 Free article. No abstract available.
Heterogeneous worldwide access and pricing of Tafamidis.
Wardhere A, Bampatsias D, Fine N, Garcia-Pavia P, Grogan M, Kristen AV, Damy T, Sekijima Y, Maurer MS. Wardhere A, et al. Among authors: kristen av. Amyloid. 2024 Mar;31(1):73-75. doi: 10.1080/13506129.2023.2263620. Epub 2023 Oct 3. Amyloid. 2024. PMID: 37787511 No abstract available.
Clinical and Genotype Characteristics and Symptom Migration in Patients With Mixed Phenotype Transthyretin Amyloidosis from the Transthyretin Amyloidosis Outcomes Survey.
González-Moreno J, Dispenzieri A, Grogan M, Coelho T, Tournev I, Waddington-Cruz M, Wixner J, Diemberger I, Garcia-Pavia P, Chapman D, Gupta P, Glass O, Amass L; THAOS investigators. González-Moreno J, et al. Cardiol Ther. 2024 Mar;13(1):117-135. doi: 10.1007/s40119-023-00344-3. Epub 2023 Dec 20. Cardiol Ther. 2024. PMID: 38117424 Free PMC article.
Age- and Sex-Related Differences in Patients With Wild-Type Transthyretin Amyloidosis: Insights From THAOS.
Mora-Ayestaran N, Dispenzieri A, Kristen AV, Maurer MS, Diemberger I, Drachman BM, Grogan M, Gupta P, Glass O, Amass L, Garcia-Pavia P; THAOS Investigators. Mora-Ayestaran N, et al. Among authors: kristen av. JACC Adv. 2024 Jul 12;3(8):101086. doi: 10.1016/j.jacadv.2024.101086. eCollection 2024 Aug. JACC Adv. 2024. PMID: 39105117 Free PMC article.
Phenotypic characteristics of F64L, I68L, I107V, and S77Y ATTRv genotypes from the Transthyretin Amyloidosis Outcomes Survey (THAOS).
Gentile L, Diemberger I, Plante-Bordeneuve V, Mazzeo A, Dori A, Luigetti M, Di Paolantonio A, Dispenzieri A, Grogan M, Waddington Cruz M, Adams D, Inamo J, Kristen AV, Lino Cirami C, Chapman D, Gupta P, Glass O, Amass L. Gentile L, et al. Among authors: kristen av. PLoS One. 2024 Jan 19;19(1):e0292435. doi: 10.1371/journal.pone.0292435. eCollection 2024. PLoS One. 2024. PMID: 38241252 Free PMC article.
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
118 results