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Page 1
Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy.
Coelho T, Marques W Jr, Dasgupta NR, Chao CC, Parman Y, França MC Jr, Guo YC, Wixner J, Ro LS, Calandra CR, Kowacs PA, Berk JL, Obici L, Barroso FA, Weiler M, Conceição I, Jung SW, Buchele G, Brambatti M, Chen J, Hughes SG, Schneider E, Viney NJ, Masri A, Gertz MR, Ando Y, Gillmore JD, Khella S, Dyck PJB, Waddington Cruz M; NEURO-TTRansform Investigators. Coelho T, et al. JAMA. 2023 Oct 17;330(15):1448-1458. doi: 10.1001/jama.2023.18688. JAMA. 2023. PMID: 37768671 Free PMC article.
Guillain-Barré Syndrome and COVID-19 Vaccine: A Multicenter Retrospective Study of 46 Cases.
Castiglione JI, Crespo JM, Bendersky M, Silveira FO, Lecchini L, Luis MB, Zambrano FC, Cotti N, Simison CJ, Aguirre F, Piedrabuena MA, Alonso RN, Azcona CL, Sosa PS, Maldonado E, Varela F, Bettini M, Rey RD, Cejas LL, Rugiero M, Reisin R, Barroso F. Castiglione JI, et al. Among authors: rugiero m. J Clin Neuromuscul Dis. 2023 Sep 1;25(1):1-10. doi: 10.1097/CND.0000000000000437. J Clin Neuromuscul Dis. 2023. PMID: 37611264
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. Amyloid. 2023 Dec;30(4):445-448. doi: 10.1080/13506129.2023.2229484. Epub 2023 Jul 17. Amyloid. 2023. PMID: 37459334 No abstract available.
Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks: A Phase 3 Randomized Clinical Trial.
Kishnani PS, Diaz-Manera J, Toscano A, Clemens PR, Ladha S, Berger KI, Kushlaf H, Straub V, Carvalho G, Mozaffar T, Roberts M, Attarian S, Chien YH, Choi YC, Day JW, Erdem-Ozdamar S, Illarioshkin S, Goker-Alpan O, Kostera-Pruszczyk A, van der Ploeg AT, An Haack K, Huynh-Ba O, Tammireddy S, Thibault N, Zhou T, Dimachkie MM, Schoser B; COMET Investigator Group. Kishnani PS, et al. JAMA Neurol. 2023 Jun 1;80(6):558-567. doi: 10.1001/jamaneurol.2023.0552. JAMA Neurol. 2023. PMID: 37036722 Free PMC article. Clinical Trial.
[Clinical Practice Guidelines for diagnosis of organic involvement of amyloidosis: Part 3/3 Year 2020].
Posadas Martinez ML, Aguirre MA, Greloni G, Marciano S, Perez de Arenaza D, Rugiero M, Tomei M, Peuchot V, Nucifora E, Aparicio LS, Leon Cejas L, Luxardo R, Popelka P, Reisin R, Seilikovivh P, Varela CF. Posadas Martinez ML, et al. Among authors: rugiero m. Rev Fac Cien Med Univ Nac Cordoba. 2022 Dec 21;79(4):391-399. doi: 10.31053/1853.0605.v79.n4.30903. Rev Fac Cien Med Univ Nac Cordoba. 2022. PMID: 36542592 Free PMC article. Spanish.
Clinical and genetic profile of patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS): 14-year update.
Dispenzieri A, Coelho T, Conceição I, Waddington-Cruz M, Wixner J, Kristen AV, Rapezzi C, Planté-Bordeneuve V, Gonzalez-Moreno J, Maurer MS, Grogan M, Chapman D, Amass L; THAOS investigators. Dispenzieri A, et al. Orphanet J Rare Dis. 2022 Jun 18;17(1):236. doi: 10.1186/s13023-022-02359-w. Orphanet J Rare Dis. 2022. PMID: 35717381 Free PMC article.
37 results