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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. Among authors: kaczmarek r. 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.
Recommendations for a minimum data set for monitoring gene therapy in hemophilia: communication from the ISTH SSC Working Group on Gene Therapy.
Miesbach W, Konkle B, Chowdary P, Kaczmarek R, Leebeek F, Mahlangu J, Makris M, Pipe SW, Srivastava A, Voorberg J, Pierce GF, Peyvandi F. Miesbach W, et al. Among authors: kaczmarek r. J Thromb Haemost. 2024 May;22(5):1510-1515. doi: 10.1016/j.jtha.2023.12.039. Epub 2024 Jan 18. J Thromb Haemost. 2024. PMID: 38242208
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A.
Mahlangu J, Kaczmarek R, von Drygalski A, Shapiro S, Chou SC, Ozelo MC, Kenet G, Peyvandi F, Wang M, Madan B, Key NS, Laffan M, Dunn AL, Mason J, Quon DV, Symington E, Leavitt AD, Oldenburg J, Chambost H, Reding MT, Jayaram K, Yu H, Mahajan R, Chavele KM, Reddy DB, Henshaw J, Robinson TM, Wong WY, Pipe SW; GENEr8-1 Trial Group. Mahlangu J, et al. Among authors: kaczmarek r. N Engl J Med. 2023 Feb 23;388(8):694-705. doi: 10.1056/NEJMoa2211075. N Engl J Med. 2023. PMID: 36812433 Clinical Trial.
Inhibitor development according to concentrate in severe hemophilia: reporting on 1392 Previously Untreated Patients from Europe and Canada.
Fischer K, Lassila R, Peyvandi F, Gatt A, Hollingsworth R, Lambert T, Kaczmarek R, Bettle A, Samji N, Rivard GÉ, Carcao M, Iorio A, Makris M; EUHASS and CHESS participants. Fischer K, et al. Among authors: kaczmarek r. Res Pract Thromb Haemost. 2023 Nov 20;7(8):102265. doi: 10.1016/j.rpth.2023.102265. eCollection 2023 Nov. Res Pract Thromb Haemost. 2023. PMID: 38193044 Free PMC article.
170 results