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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
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.
Looking to the future of gene therapy for hemophilia A and B.
Kaczmarek R, Herzog RW. Kaczmarek R, et al. Expert Rev Hematol. 2023 Jul-Dec;16(11):807-809. doi: 10.1080/17474086.2023.2268279. Epub 2023 Nov 17. Expert Rev Hematol. 2023. PMID: 37798911 No abstract available.
170 results