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1
Making friends out of foes: novel therapeutic approaches for haemophilia.化敌为友:血友病的新型治疗方法
Blood Transfus. 2021 Mar;19(2):179-180. doi: 10.2450/2020.0403-20. Epub 2020 Dec 23.
2
Recent advances in developing specific therapies for haemophilia.血友病特异性治疗方法的最新进展。
Br J Haematol. 2018 Apr;181(2):161-172. doi: 10.1111/bjh.15084. Epub 2018 Jan 23.
3
Light and shadows of the new therapies for haemophilia treatment in the COVID-19 era.新冠疫情时代血友病治疗新疗法的利弊
Blood Transfus. 2021 Jan;19(1):73-76. doi: 10.2450/2020.0241-20. Epub 2020 Sep 18.
4
Antibodies in the Treatment of Haemophilia A-A Biochemical Perspective.抗血友病 A 治疗中的抗体——生物化学视角。
Hamostaseologie. 2019 Feb;39(1):36-41. doi: 10.1055/s-0038-1677521. Epub 2019 Jan 29.
5
When innovation goes fast. The case of hemophilia.当创新步伐加快。以血友病为例。
Curr Opin Pharmacol. 2019 Apr;45:95-101. doi: 10.1016/j.coph.2019.06.005. Epub 2019 Jul 4.
6
Emerging Therapeutic Strategies in the Treatment of Hemophilia A.治疗甲型血友病的新兴治疗策略
Semin Thromb Hemost. 2017 Sep;43(6):581-590. doi: 10.1055/s-0037-1604053. Epub 2017 Jul 27.
7
Therapeutic choices in persons with haemophilia at the time of COVID-19.新冠疫情期间血友病患者的治疗选择
Blood Transfus. 2020 Jul;18(4):326-327. doi: 10.2450/2020.0154-20.
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The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing.血友病治疗领域的变化:从标准半衰期凝血因子浓缩物到基因编辑。
Lancet. 2021 Feb 13;397(10274):630-640. doi: 10.1016/S0140-6736(20)32722-7. Epub 2021 Jan 15.
9
Curing Hemophilia: Repeated Treatments versus a One-Off Fix.治愈血友病:重复治疗与一次性修复
Mol Ther. 2020 May 6;28(5):1229-1230. doi: 10.1016/j.ymthe.2020.04.012. Epub 2020 Apr 17.
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Confronting COVID-19: Issues in Hemophilia and Congenital Bleeding Disorders.直面新冠疫情:血友病和先天性出血性疾病相关问题
Semin Thromb Hemost. 2020 Oct;46(7):819-822. doi: 10.1055/s-0040-1712961. Epub 2020 Jun 8.

本文引用的文献

1
Gene therapy to cure haemophilia: Is robust scientific inquiry the missing factor?基因疗法治疗血友病:有力的科学探究是否是缺失的因素?
Haemophilia. 2020 Nov;26(6):931-933. doi: 10.1111/hae.14131. Epub 2020 Sep 10.
2
Gene therapy for hemophilia: anticipating the unexpected.血友病的基因治疗:预见到意外情况
Blood Adv. 2020 Aug 11;4(15):3788. doi: 10.1182/bloodadvances.2020002142.
3
Bispecific Antibodies and Advances in Non-Gene Therapy Options in Hemophilia.双特异性抗体与血友病非基因治疗选择的进展
Res Pract Thromb Haemost. 2020 Apr 28;4(4):446-454. doi: 10.1002/rth2.12337. eCollection 2020 May.
4
Hemophilia therapy: the future has begun.血友病治疗:未来已来。
Haematologica. 2020 Mar;105(3):545-553. doi: 10.3324/haematol.2019.232132. Epub 2020 Feb 14.
5
Tolerating Factor VIII: Recent Progress.耐受因子 VIII:最新进展。
Front Immunol. 2020 Jan 10;10:2991. doi: 10.3389/fimmu.2019.02991. eCollection 2019.
6
Safety and efficacy of emicizumab and other novel agents in newborns and infants.艾美赛珠单抗及其他新型药物在新生儿和婴儿中的安全性与有效性。
Haemophilia. 2019 Sep;25(5):e334-e335. doi: 10.1111/hae.13822. Epub 2019 Jul 30.
7
Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study.每4周给予艾美赛珠单抗预防治疗A型血友病患者的疗效、安全性及药代动力学(HAVEN 4):一项多中心、开放标签、非随机3期研究
Lancet Haematol. 2019 Jun;6(6):e295-e305. doi: 10.1016/S2352-3026(19)30054-7. Epub 2019 Apr 16.
8
Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate.儿童和成人的初级和挽救性免疫耐受诱导:一项含有 VWF 的血浆源性 FVIII 浓缩物的多中心国际研究。
Haemophilia. 2014 Jan;20(1):83-91. doi: 10.1111/hae.12263. Epub 2013 Nov 19.
9
The best of times, the worst of times: a story of haemophilia.最好的时代,最坏的时代:一个血友病的故事。
Clin Med (Lond). 2009 Oct;9(5):453-8. doi: 10.7861/clinmedicine.9-5-453.
10
Consideration in hemophilia therapy selection.血友病治疗方案选择时的考量因素。
Semin Hematol. 2006 Apr;43(2 Suppl 3):S23-7. doi: 10.1053/j.seminhematol.2006.02.002.

Making friends out of foes: novel therapeutic approaches for haemophilia.

作者信息

Mactier Catriona, Hermans Cedric

机构信息

Great Ormond Hospital for Children NHS Trust Haemophilia Centre, London, United Kingdom.

Division of Haematology, Haemophilia Centre, Saint-Luc University Hospital, Université catholique de Louvain (UCLouvain), Brussels, Belgium.

出版信息

Blood Transfus. 2021 Mar;19(2):179-180. doi: 10.2450/2020.0403-20. Epub 2020 Dec 23.

DOI:10.2450/2020.0403-20
PMID:33656979
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7925227/
Abstract
摘要