Advances and innovations in haemophilia treatment
| Autor: | Harris T J R, Rob Peters |
|---|---|
| Rok vydání: | 2018 |
| Předmět: |
0301 basic medicine
congenital hereditary and neonatal diseases and abnormalities medicine.medical_specialty Standard of care Disease 030204 cardiovascular system & hematology Hemophilia A Haemophilia 03 medical and health sciences 0302 clinical medicine hemic and lymphatic diseases Drug Discovery medicine Animals Humans Intensive care medicine Pharmacology Clotting factor Modalities business.industry Anticoagulants Treatment options General Medicine medicine.disease Blood Coagulation Factors 030104 developmental biology business |
| Zdroj: | Nature Reviews Drug Discovery. 17:493-508 |
| ISSN: | 1474-1784 1474-1776 |
| DOI: | 10.1038/nrd.2018.70 |
| Popis: | New therapies in development for haemophilia — including gene therapies, proteins with extended half-lives and inhibitors of natural anticoagulants — look poised to change the management of this disease. In this Review, Peters and Harris discuss recent progress, including the hurdles that still need to be overcome in order to translate these advances into routine clinical use. Haemophilia is a rare disease for which the approved therapeutic options have remained virtually unchanged for 50 years. In the past decade, however, there has been an explosion of innovation in the treatment options that are either in development or have been approved for haemophilia, including engineered clotting factors and an extensive pipeline of new approaches and modalities. Several of these new modalities, especially gene therapy, demonstrate proof of principle in haemophilia but could have broader applications. These advances, in combination with better diagnostics, are now enabling clinicians to improve the standard of care for people with haemophilia. The different mechanisms of action and modifications used in these therapies have implications for their safe and efficacious use, which must be balanced with their therapeutic utility. This Review focuses on the biological aspects of the most advanced and innovative approaches for haemophilia treatment and considers their future use. |
| Databáze: | OpenAIRE |
| Externí odkaz: |
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