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Page 1
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial.
Coppens M, Pipe SW, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan PE, Leebeek FWG; HOPE-B Investigators. Coppens M, et al. Lancet Haematol. 2024 Apr;11(4):e265-e275. doi: 10.1016/S2352-3026(24)00006-1. Epub 2024 Mar 1. Lancet Haematol. 2024. PMID: 38437857 Clinical Trial.
Multicenter assessment and longitudinal study of the prevalence of antibodies and related adaptive immune responses to AAV in adult males with hemophilia.
Pabinger I, Ayash-Rashkovsky M, Escobar M, Konkle BA, Mingot-Castellano ME, Mullins ES, Negrier C, Pan L, Rajavel K, Yan B, Chapin J. Pabinger I, et al. Among authors: konkle ba. Gene Ther. 2024 May;31(5-6):273-284. doi: 10.1038/s41434-024-00441-5. Epub 2024 Feb 14. Gene Ther. 2024. PMID: 38355967 Free PMC article.
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: konkle b. 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
The World Federation of Hemophilia World Bleeding Disorders Registry: insights from the first 10,000 patients.
Coffin D, Gouider E, Konkle B, Hermans C, Lambert C, Diop S, Ayoub E, Tootoonchian E, Youttananukorn T, Dakik P, Pereira T, Iorio A, Pierce GF; World Bleeding Disorders Registry Participating Investigators. Coffin D, et al. Among authors: konkle b. Res Pract Thromb Haemost. 2023 Nov 20;7(8):102264. doi: 10.1016/j.rpth.2023.102264. eCollection 2023 Nov. Res Pract Thromb Haemost. 2023. PMID: 38193052 Free PMC article.
Landmark endorsement of a global registry: The European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP), publicly endorses World Federation of Hemophilia Gene Therapy Registry as global standard.
Konkle BA, Peyvandi F, Coffin D, Naccache M, Youttananukorn T, Pierce GF; WFH Gene Therapy Registry Scientific Advisory Board. Konkle BA, et al. Haemophilia. 2024 Jan;30(1):232-235. doi: 10.1111/hae.14912. Epub 2023 Dec 18. Haemophilia. 2024. PMID: 38111095 No abstract available.
Management of pregnant women who have bleeding disorders.
James AH, Pacheco LD, Konkle BA. James AH, et al. Among authors: konkle ba. Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):229-236. doi: 10.1182/hematology.2023000475. Hematology Am Soc Hematol Educ Program. 2023. PMID: 38066866
Restrictive or Liberal Transfusion Strategy in Myocardial Infarction and Anemia.
Carson JL, Brooks MM, Hébert PC, Goodman SG, Bertolet M, Glynn SA, Chaitman BR, Simon T, Lopes RD, Goldsweig AM, DeFilippis AP, Abbott JD, Potter BJ, Carrier FM, Rao SV, Cooper HA, Ghafghazi S, Fergusson DA, Kostis WJ, Noveck H, Kim S, Tessalee M, Ducrocq G, de Barros E Silva PGM, Triulzi DJ, Alsweiler C, Menegus MA, Neary JD, Uhl L, Strom JB, Fordyce CB, Ferrari E, Silvain J, Wood FO, Daneault B, Polonsky TS, Senaratne M, Puymirat E, Bouleti C, Lattuca B, White HD, Kelsey SF, Steg PG, Alexander JH; MINT Investigators. Carson JL, et al. N Engl J Med. 2023 Dec 28;389(26):2446-2456. doi: 10.1056/NEJMoa2307983. Epub 2023 Nov 11. N Engl J Med. 2023. PMID: 37952133 Clinical Trial.
A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories.
Pipe S, Sadeghi-Khomami A, Konkle BA, Kitchen S, Negrier C, Liu M, Santagostino E, Willemze A, Abad-Franch L, Knobe K, Seth Chhabra E. Pipe S, et al. Among authors: konkle ba. Haemophilia. 2024 Jan;30(1):214-223. doi: 10.1111/hae.14831. Epub 2023 Oct 30. Haemophilia. 2024. PMID: 37902390
A Scan of Pleiotropic Immune Mediated Disease Genes Identifies Novel Determinants of Baseline FVIII Inhibitor Status in Hemophilia-A.
Howard T, Almieda M, Diego V, Viel K, Luu B, Haack K, Raja R, Ameri A, Chitlur M, Rydz N, Lillicrap D, Watts R, Kessler C, Ramsey C, Dinh L, Kim B, Powell J, Peralta J, Bouls R, Abraham S, Shen YM, Murillo C, Mead H, Lehmann P, Fine E, Escobar M, Kumar S, Williams-Blangero S, Kasper C, Almasy L, Cole S, Blangero J, Konkle B. Howard T, et al. Among authors: konkle b. Res Sq [Preprint]. 2023 Oct 18:rs.3.rs-3371095. doi: 10.21203/rs.3.rs-3371095/v1. Res Sq. 2023. PMID: 37886476 Free PMC article. Preprint.
234 results