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Page 1
Minimal manifestation status and prednisone withdrawal in the MGTX trial.
Lee I, Kuo HC, Aban IB, Cutter GR, McPherson T, Kaminski HJ, Sussman J, Ströbel P, Oger J, Cea G, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BRF, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Verschuuren JJG, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Minisman G, Sonett JR, Wolfe GI; MGTX study group. Lee I, et al. Neurology. 2020 Aug 11;95(6):e755-e766. doi: 10.1212/WNL.0000000000010031. Epub 2020 Jul 1. Neurology. 2020. PMID: 32611638 Free PMC article. 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: waddington cruz m. Amyloid. 2023 Dec;30(4):445-448. doi: 10.1080/13506129.2023.2229484. Epub 2023 Jul 17. Amyloid. 2023. PMID: 37459334 No abstract available.
Liver-directed drugs for transthyretin-mediated amyloidosis.
Brannagan TH 3rd, Berk JL, Gillmore JD, Maurer MS, Waddington-Cruz M, Fontana M, Masri A, Obici L, Brambatti M, Baker BF, Hannan LA, Buchele G, Viney NJ, Coelho T, Nativi-Nicolau J. Brannagan TH 3rd, et al. J Peripher Nerv Syst. 2022 Dec;27(4):228-237. doi: 10.1111/jns.12519. Epub 2022 Nov 16. J Peripher Nerv Syst. 2022. PMID: 36345805 Free PMC article. Review.
A multicentric study of the disease risks and first manifestations in hereditary transthyretin amyloidosis (ATTRv): insights for an earlier diagnosis.
Planté-Bordeneuve V, Gorram F, Olsson M, Anan I, Mazzeo A, Gentile L, Cisneros-Barroso E, Gonzalez-Moreno J, Losada I, Waddington-Cruz M, Pinto LF, Parman Y, Fanen P, Alarcon F, Nuel G. Planté-Bordeneuve V, et al. Amyloid. 2023 Sep;30(3):313-320. doi: 10.1080/13506129.2023.2178891. Epub 2023 Feb 21. Amyloid. 2023. PMID: 36994840
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: waddington cruz m. PLoS One. 2024 Jan 19;19(1):e0292435. doi: 10.1371/journal.pone.0292435. eCollection 2024. PLoS One. 2024. PMID: 38241252 Free PMC article.
Brazilian consensus for diagnosis, management and treatment of hereditary transthyretin amyloidosis with peripheral neuropathy: second edition.
Pinto MV, França MC Jr, Gonçalves MVM, Machado-Costa MC, Freitas MRG, Gondim FAA, Marrone CD, Martinez ARM, Moreira CL, Nascimento OJM, Covaleski APP, Oliveira ASB, Pupe CCB, Rodrigues MMJ, Rotta FT, Scola RH, Marques W Jr, Waddington-Cruz M. Pinto MV, et al. Arq Neuropsiquiatr. 2023 Mar;81(3):308-321. doi: 10.1055/s-0043-1764412. Epub 2023 Apr 14. Arq Neuropsiquiatr. 2023. PMID: 37059440 Free PMC article.
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. Among authors: waddington cruz m. 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.
A 15-year consolidated overview of data in over 6000 patients from the Transthyretin Amyloidosis Outcomes Survey (THAOS).
Gentile L, Coelho T, Dispenzieri A, Conceição I, Waddington-Cruz M, Kristen A, Wixner J, Diemberger I, Gonzalez-Moreno J, Cariou E, Maurer MS, Planté-Bordeneuve V, Garcia-Pavia P, Tournev I, Gonzalez-Costello J, Duarte AG, Grogan M, Mazzeo A, Chapman D, Gupta P, Glass O, Amass L; THAOS investigators. Gentile L, et al. Among authors: waddington cruz m. Orphanet J Rare Dis. 2023 Nov 10;18(1):350. doi: 10.1186/s13023-023-02962-5. Orphanet J Rare Dis. 2023. PMID: 37946256 Free PMC article.
Randomized Trial of Thymectomy in Myasthenia Gravis.
Wolfe GI, Kaminski HJ, Aban IB, Minisman G, Kuo HC, Marx A, Ströbel P, Mazia C, Oger J, Cea JG, Heckmann JM, Evoli A, Nix W, Ciafaloni E, Antonini G, Witoonpanich R, King JO, Beydoun SR, Chalk CH, Barboi AC, Amato AA, Shaibani AI, Katirji B, Lecky BR, Buckley C, Vincent A, Dias-Tosta E, Yoshikawa H, Waddington-Cruz M, Pulley MT, Rivner MH, Kostera-Pruszczyk A, Pascuzzi RM, Jackson CE, Garcia Ramos GS, Verschuuren JJ, Massey JM, Kissel JT, Werneck LC, Benatar M, Barohn RJ, Tandan R, Mozaffar T, Conwit R, Odenkirchen J, Sonett JR, Jaretzki A 3rd, Newsom-Davis J, Cutter GR; MGTX Study Group. Wolfe GI, et al. N Engl J Med. 2016 Aug 11;375(6):511-22. doi: 10.1056/NEJMoa1602489. N Engl J Med. 2016. PMID: 27509100 Free PMC article. Clinical Trial.
56 results