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Disruptive technology and hemophilia care: The multiple impacts of emicizumab
Emicizumab, a bispecific antibody mimicking the action of factor VIII (FVIII), is currently the first and only approved and increasingly accessible disruptive treatment option for hemophilia A, a disease so far mainly treated with frequent intravenous infusions of FVIII concentrates or bypassing age...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8116836/ https://www.ncbi.nlm.nih.gov/pubmed/34027289 http://dx.doi.org/10.1002/rth2.12508 |
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author | Hermans, Cedric Makris, Mike |
author_facet | Hermans, Cedric Makris, Mike |
author_sort | Hermans, Cedric |
collection | PubMed |
description | Emicizumab, a bispecific antibody mimicking the action of factor VIII (FVIII), is currently the first and only approved and increasingly accessible disruptive treatment option for hemophilia A, a disease so far mainly treated with frequent intravenous infusions of FVIII concentrates or bypassing agents in case of inhibitor development. Other disruptive treatments are expected to follow, such as agents that rebalance coagulation and gene therapy with the ambition of curing hemophilia. While these treatment options represent major achievements or expectations, their adoption and implementation should consider their multiple direct and indirect, immediate or delayed, consequences on hemophilia care globally. It is these multiple changes, present and future, already visible or hypothetical, that this article intends to review and explore. |
format | Online Article Text |
id | pubmed-8116836 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | John Wiley and Sons Inc. |
record_format | MEDLINE/PubMed |
spelling | pubmed-81168362021-05-20 Disruptive technology and hemophilia care: The multiple impacts of emicizumab Hermans, Cedric Makris, Mike Res Pract Thromb Haemost Forum Emicizumab, a bispecific antibody mimicking the action of factor VIII (FVIII), is currently the first and only approved and increasingly accessible disruptive treatment option for hemophilia A, a disease so far mainly treated with frequent intravenous infusions of FVIII concentrates or bypassing agents in case of inhibitor development. Other disruptive treatments are expected to follow, such as agents that rebalance coagulation and gene therapy with the ambition of curing hemophilia. While these treatment options represent major achievements or expectations, their adoption and implementation should consider their multiple direct and indirect, immediate or delayed, consequences on hemophilia care globally. It is these multiple changes, present and future, already visible or hypothetical, that this article intends to review and explore. John Wiley and Sons Inc. 2021-05-07 /pmc/articles/PMC8116836/ /pubmed/34027289 http://dx.doi.org/10.1002/rth2.12508 Text en © 2021 The Authors. Research and Practice in Thrombosis and Haemostasis published by Wiley Periodicals LLC on behalf of International Society on Thrombosis and Haemostasis (ISTH). https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the terms of the http://creativecommons.org/licenses/by-nc-nd/4.0/ (https://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made. |
spellingShingle | Forum Hermans, Cedric Makris, Mike Disruptive technology and hemophilia care: The multiple impacts of emicizumab |
title | Disruptive technology and hemophilia care: The multiple impacts of emicizumab |
title_full | Disruptive technology and hemophilia care: The multiple impacts of emicizumab |
title_fullStr | Disruptive technology and hemophilia care: The multiple impacts of emicizumab |
title_full_unstemmed | Disruptive technology and hemophilia care: The multiple impacts of emicizumab |
title_short | Disruptive technology and hemophilia care: The multiple impacts of emicizumab |
title_sort | disruptive technology and hemophilia care: the multiple impacts of emicizumab |
topic | Forum |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8116836/ https://www.ncbi.nlm.nih.gov/pubmed/34027289 http://dx.doi.org/10.1002/rth2.12508 |
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