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Page 1
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
World Federation of Hemophilia Gene Therapy Registry.
Konkle BA, Coffin D, Pierce GF, Clark C, George L, Iorio A, Mahlangu J, Naccache M, O'Mahony B, Peyvandi F, Pipe S, Quartel A, Sawyer EK, Skinner MW, Tortella B, Watson C, Winburn I; Members of the WFH Gene Therapy Registery Steering Committee. Konkle BA, et al. Haemophilia. 2020 Jul;26(4):563-564. doi: 10.1111/hae.14015. Epub 2020 May 27. Haemophilia. 2020. PMID: 32462720 Free PMC article. No abstract available.
Kreuth V initiative: European consensus proposals for treatment of hemophilia using standard products, extended half-life coagulation factor concentrates and non-replacement therapies.
Peyvandi F, Berger K, Seitz R, Hilger A, Hecquet ML, Wierer M, Buchheit KH, O'Mahony B, Bok A, Makris M, Mansmann U, Schramm W, Mannucci PM. Peyvandi F, et al. Haematologica. 2020 Aug;105(8):2038-2043. doi: 10.3324/haematol.2019.242735. Epub 2020 May 28. Haematologica. 2020. PMID: 32467138 Free PMC article.
Vaccination against COVID-19: Rationale, modalities and precautions for patients with haemophilia and other inherited bleeding disorders.
Kaczmarek R, El Ekiaby M, Hart DP, Hermans C, Makris M, Noone D, O'Mahony B, Page D, Peyvandi F, Pipe SW, Sannié T, Schlenkrich U, Skinner MW, Srivastava A, Bok A, Pierce GF; World Federation of Hemophilia (WFH), European Association for Haemophilia, Allied Disorders (EAHAD), European Haemophilia Consortium (EHC), U.S. National Hemophilia Foundation (NHF). Kaczmarek R, et al. Haemophilia. 2021 Jul;27(4):515-518. doi: 10.1111/hae.14271. Epub 2021 Mar 2. Haemophilia. 2021. PMID: 33651911 Free PMC article. No abstract available.
Eliminating Panglossian thinking in development of AAV therapeutics.
Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Skinner MW. Kaczmarek R, et al. Mol Ther. 2021 Dec 1;29(12):3325-3327. doi: 10.1016/j.ymthe.2021.10.025. Epub 2021 Nov 10. Mol Ther. 2021. PMID: 34758292 Free PMC article. No abstract available.
Pain and functional disability amongst adults with moderate and severe haemophilia from the Irish personalised approach to the treatment of haemophilia (iPATH) study.
Kennedy M, O' Mahony B, Roche S, McGowan M, Singleton E, Ryan K, O' Connell NM, Pipe SW, Lavin M, O' Donnell JS, Turecek PL, Gormley J; on behalf of the iPATH study group. Kennedy M, et al. Eur J Haematol. 2022 Jun;108(6):518-527. doi: 10.1111/ejh.13763. Epub 2022 Mar 30. Eur J Haematol. 2022. PMID: 35258118 Free PMC article.
Hemophilia gene therapy: first, do no harm.
Valentino LA, Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Rotellini D, Skinner MW. Valentino LA, et al. J Thromb Haemost. 2023 Sep;21(9):2354-2361. doi: 10.1016/j.jtha.2023.06.016. Epub 2023 Jun 21. J Thromb Haemost. 2023. PMID: 37353081 Free article. Review.
113 results