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Page 1
Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults.
Konkle BA, Oldenburg J, Pasi J, Kulkarni R, Nolan B, Mahlangu J, Young G, Brown SA, Pabinger I, Shapiro A, Négrier C, Blanchette V, Ragni MV, Dumont J, Lethagen S. Konkle BA, et al. Among authors: negrier c. Res Pract Thromb Haemost. 2023 Aug 22;7(6):102180. doi: 10.1016/j.rpth.2023.102180. eCollection 2023 Aug. Res Pract Thromb Haemost. 2023. PMID: 37753224 Free PMC article.
Pharmacokinetics and coagulation biomarkers in children and adults with hemophilia A receiving emicizumab prophylaxis every 1, 2, or 4 weeks.
Kiialainen A, Adamkewicz JI, Petry C, Oldenburg J, Pipe SW, Young G, Mahlangu J, Lehle M, Niggli M, Castaman G, Jiménez-Yuste V, Shima M, Négrier C, Schmitt C. Kiialainen A, et al. Among authors: negrier c. Res Pract Thromb Haemost. 2023 Dec 29;8(1):102306. doi: 10.1016/j.rpth.2023.102306. eCollection 2024 Jan. Res Pract Thromb Haemost. 2023. PMID: 38282901 Free PMC article.
Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B.
Cuker A, Kavakli K, Frenzel L, Wang JD, Astermark J, Cerqueira MH, Iorio A, Katsarou-Fasouli O, Klamroth R, Shapiro AD, Hermans C, Ishiguro A, Leavitt AD, Oldenburg JB, Ozelo MC, Teitel J, Biondo F, Fang A, Fuiman J, McKay J, Sun P, Rasko JEJ, Rupon J; BENEGENE-2 Trial Investigators. Cuker A, et al. N Engl J Med. 2024 Sep 26;391(12):1108-1118. doi: 10.1056/NEJMoa2302982. N Engl J Med. 2024. PMID: 39321362 Clinical Trial.
Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial.
Kenet G, Nolan B, Zulfikar B, Antmen B, Kampmann P, Matsushita T, You CW, Vilchevska K, Bagot CN, Sharif A, Peyvandi F, Young G, Negrier C, Chi J, Kittner B, Sussebach C, Shammas F, Mei B, Andersson S, Kavakli K. Kenet G, et al. Among authors: negrier c. Blood. 2024 May 30;143(22):2256-2269. doi: 10.1182/blood.2023021864. Blood. 2024. PMID: 38452197 Free PMC article. 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: negrier c. 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.
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: negrier c. Haemophilia. 2024 Jan;30(1):214-223. doi: 10.1111/hae.14831. Epub 2023 Oct 30. Haemophilia. 2024. PMID: 37902390
Determinants of adherence and consequences of the transition from adolescence to adulthood among young people with severe haemophilia (TRANSHEMO): A multicentric French national observational cross-sectional study based on the FranceCoag registry.
Nguyen NAT, Auquier P, Beltran Anzola A, Harroche A, Castet S, Huguenin Y, Meunier S, Repesse Y, D'Oiron R, Rauch A, Desprez D, Spiegel A, Chamouni P, Schneider P, Baumstarck K, Boucekine M, Tabele C, Viprey M, Leroy T, Roques MA, Sannie T, Giraud N, Chambost H, Resseguier N; TRANSHEMO Study group. Nguyen NAT, et al. Haemophilia. 2023 Sep;29(5):1202-1218. doi: 10.1111/hae.14841. Epub 2023 Aug 12. Haemophilia. 2023. PMID: 37572328
289 results