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Journal article
Emerging and future therapies for hemophilia
Journal of blood medicine, v 6, pp 245-255
03 Sep 2015
PMID: 26366108
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
The evolution of care in hemophilia is a remarkable story. Over the last 60 years, advances in protein purification, protein chemistry, donor screening, viral inactivation, gene sequencing, gene cloning, and recombinant protein production have dramatically enhanced the treatment and lives of patients with hemophilia. Recent efforts have produced enhanced half-life (EHL) clotting factors to better support prophylaxis and decrease the frequency of infusions. Medical needs remain in the areas of alternate modes of administration to decrease the need for venous access, better treatment, and prophylaxis for patients who form antibodies to clotting factors, and ultimately a cure of the underlying genetic defect. In this brief review, the authors summarize data on EHL clotting factors, introduce agents whose mode of action is not clotting factor replacement, and list current gene therapy efforts.
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Details
- Title
- Emerging and future therapies for hemophilia
- Creators
- Marcus E Carr - Pfizer (United States)Bartholomew J Tortella - Drexel University College of Medicine, Philadelphia, PA, USA ; Global Innovative Pharma, Pfizer, Inc., Collegeville, PA, USA.
- Publication Details
- Journal of blood medicine, v 6, pp 245-255
- Publisher
- Dove Medical Press
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Engineering Technology
- Web of Science ID
- WOS:000213911600026
- Scopus ID
- 2-s2.0-85006216296
- Other Identifier
- 991019173833904721
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- Collaboration types
- Industry collaboration
- Domestic collaboration
- Web of Science research areas
- Hematology