Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved]
Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change he...
| Main Authors: | , , |
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| Format: | Article |
| Language: | English |
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F1000 Research Ltd
2018-04-01
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| Series: | F1000Research |
| Online Access: | https://f1000research.com/articles/7-489/v1 |
| _version_ | 1828175840978927616 |
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| author | Valder R. Arruda Bhavya S. Doshi Benjamin J. Samelson-Jones |
| author_facet | Valder R. Arruda Bhavya S. Doshi Benjamin J. Samelson-Jones |
| author_sort | Valder R. Arruda |
| collection | DOAJ |
| description | Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category. |
| first_indexed | 2024-04-12T04:34:19Z |
| format | Article |
| id | doaj.art-2f328eb1aaa84f4e8e5929c68ea5fcb2 |
| institution | Directory Open Access Journal |
| issn | 2046-1402 |
| language | English |
| last_indexed | 2024-04-12T04:34:19Z |
| publishDate | 2018-04-01 |
| publisher | F1000 Research Ltd |
| record_format | Article |
| series | F1000Research |
| spelling | doaj.art-2f328eb1aaa84f4e8e5929c68ea5fcb22022-12-22T03:47:51ZengF1000 Research LtdF1000Research2046-14022018-04-01710.12688/f1000research.12491.113525Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved]Valder R. Arruda0Bhavya S. Doshi1Benjamin J. Samelson-Jones2The Children's Hospital of Philadelphia, Philadelphia, PA, USAThe Children's Hospital of Philadelphia, Philadelphia, PA, USAThe Children's Hospital of Philadelphia, Philadelphia, PA, USASeveral new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category.https://f1000research.com/articles/7-489/v1 |
| spellingShingle | Valder R. Arruda Bhavya S. Doshi Benjamin J. Samelson-Jones Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved] F1000Research |
| title | Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved] |
| title_full | Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved] |
| title_fullStr | Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved] |
| title_full_unstemmed | Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved] |
| title_short | Emerging therapies for hemophilia: controversies and unanswered questions [version 1; referees: 4 approved] |
| title_sort | emerging therapies for hemophilia controversies and unanswered questions version 1 referees 4 approved |
| url | https://f1000research.com/articles/7-489/v1 |
| work_keys_str_mv | AT valderrarruda emergingtherapiesforhemophiliacontroversiesandunansweredquestionsversion1referees4approved AT bhavyasdoshi emergingtherapiesforhemophiliacontroversiesandunansweredquestionsversion1referees4approved AT benjaminjsamelsonjones emergingtherapiesforhemophiliacontroversiesandunansweredquestionsversion1referees4approved |