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新型治疗方案的研发为血友病患者带来希望。

Development of novel treatment options for patients with haemophilia.

机构信息

Prof. Dr. Armin Reininger, Baxter Innovations GmbH, Industriestraße 67, 1220 Vienna, Austria, E-mail:

出版信息

Hamostaseologie. 2013;33 Suppl 1:S36-8.

PMID:24169902
Abstract

UNLABELLED

Treatment of haemophilia has vastly improved over the last years, but many needs are still unmet. Baxter is continuously pursuing the aim to provide new therapeutic options to patients with haemophilia and to their treating physicians. In fact, there are several opportunities to improve existing therapies, e.g., by new indications for existing products, the introduction of new products, and by novel therapeutic approaches other than factor replacement. Among these, Baxter is working on a number of innovations, such as pharmacokinetics-tailored factor VIII prophylaxis, bypassing agent prophylaxis with FEIBA in inhibitor patients, development of a longer acting pegylated recombinant FVIII, a new recombinant factor IX, a new recombinant factor FVIIa, the first recombinant von Willebrand factor, recombinant ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13) as well as gene therapy to cure haemophilia B.

CONCLUSION

Baxter is truly committed to the benefit for the patient, and therefore engaged in providing a more and more individualized treatment, in increasing efficiency of current products, in developing new products and new approaches with added value.

摘要

未标注

过去几年中,血友病的治疗已经取得了巨大的进步,但仍有许多需求未得到满足。百特公司一直致力于为血友病患者及其治疗医生提供新的治疗选择。事实上,有几种方法可以改进现有的治疗方法,例如为现有产品增加新的适应证,引入新产品,以及采用除因子替代物以外的新型治疗方法。其中,百特公司正在研究许多创新方法,例如根据药代动力学调整的因子 VIII 预防治疗,在抑制剂患者中使用 FEIBA 进行旁路激活剂预防治疗,开发一种作用时间更长的聚乙二醇化重组 FVIII、一种新型重组因子 IX、一种新型重组因子 FVIIa、首个重组 vWF、重组 ADAMTS13(一种带有血小板反应素 1 型基序的解整合素金属蛋白酶 13)以及基因治疗以治愈乙型血友病。

结论

百特公司真正致力于为患者谋福利,因此致力于提供越来越个性化的治疗方案,提高现有产品的效率,开发具有附加值的新产品和新方法。

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