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Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B.
N Engl J Med. 2023 Feb 23;388(8):706-718. doi: 10.1056/NEJMoa2211644.
N Engl J Med. 2023.
PMID: 36812434
Clinical Trial.
[Rare manifestations of monoclonal gammopathies: About two clinical cases and literature review].
Perlot Q, Hermans C, Vekemans MC.
Perlot Q, et al.
Rev Med Interne. 2023 Jun;44(6):307-310. doi: 10.1016/j.revmed.2023.03.005. Epub 2023 Mar 31.
Rev Med Interne. 2023.
PMID: 37005096
Review.
French.
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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.
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Phase 1, single-dose escalating study of marzeptacog alfa (activated), a recombinant factor VIIa variant, in patients with severe hemophilia.
Gruppo RA, Malan D, Kapocsi J, Nemes L, Hay CRM, Boggio L, Chowdary P, Tagariello G, von Drygalski A, Hua F, Scaramozza M, Arkin S; Marzeptacog alfa (activated) Study Group Investigators.
Gruppo RA, et al.
J Thromb Haemost. 2018 Oct;16(10):1984-1993. doi: 10.1111/jth.14247. Epub 2018 Aug 27.
J Thromb Haemost. 2018.
PMID: 30151972
Free article.
Clinical Trial.
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Rebuttal: accidental overdosage of recombinant factor VIIa due to mistaken package size: an inappropriate case report.
Hermans CR, Deneys V.
Hermans CR, et al.
Thromb Haemost. 2004 Dec;92(6):1455-6; author reply 1456.
Thromb Haemost. 2004.
PMID: 15583757
No abstract available.
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