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Medientyp:
E-Artikel
Titel:
Recent advances in developing specific therapies for haemophilia
Beteiligte:
Ling, Gavin;
Nathwani, Amit C.;
Tuddenham, Edward G. D.
Erschienen:
Wiley, 2018
Erschienen in:British Journal of Haematology
Sprache:
Englisch
DOI:
10.1111/bjh.15084
ISSN:
0007-1048;
1365-2141
Entstehung:
Anmerkungen:
Beschreibung:
<jats:title>Summary</jats:title><jats:p>Haemophilia therapy has undergone very rapid evolution in the last 10 years. The major limitation of current replacement therapy is the short half‐life of factors <jats:styled-content style="fixed-case">VIII</jats:styled-content> and <jats:styled-content style="fixed-case">IX</jats:styled-content>. These half‐lives have been extended by the addition of various moieties, allowing less frequent infusion regimens. Entirely novel approaches have also entered the clinic, including a bispecific antibody that mimics factor <jats:styled-content style="fixed-case">VIII</jats:styled-content> and strategies that rebalance the haemostatic mechanism by reducing antithrombin through inhibition of synthesis. These two treatments are available by subcutaneous injection at infrequent intervals and both can be used in patients with neutralising antibodies (inhibitors). Finally, a cure may be on the horizon with preliminary evidence of success for gene therapy in haemophilia B and A.</jats:p>