Hemophilia A future or investigational therapies: Difference between revisions
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{{Hemophilia A}} | {{Hemophilia A}} {{FNY}} | ||
==Overview== | |||
Research is underway into novel therapies to help in the management and possible cure of hemophilia A. | |||
== | ==Gene therapy== | ||
The goal of gene therapy is to correct the chromosomal deficit which results in Hemophilia A. Currently, there is no role for gene therapy in the management of hemophilia A, though the issue is being pursued in clinical trials. <ref> Konkle BA, Josephson NC, Nakaya Fletcher S. Hemophilia A. 2000 Sep 21 [Updated 2014 Jun 5]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. Available from: http://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/books/NBK1404/ </ref> Researchers are also studying the genomes of patients affected with inhibitors to determine why some patients develop inhibitors and others do not. Understanding a patient's genetic profile may help physicians predict a patient's risk for developing inhibitors to factor concentrates, and help guide their treatment. <ref> Why We Do research on Hemophilia | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/research.html. Accessed on Sept 20, 2016 </ref> | |||
==Future and Investigational Therapies== | |||
==References== | ==References== | ||
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[[Category:Disease]] | [[Category:Disease]] | ||
[[Category:Hematology]] | [[Category:Hematology]] | ||
[[Category:Pediatrics]] | [[Category:Pediatrics]] | ||
[[Category:Neonatology]] | [[Category:Neonatology]] | ||
Revision as of 01:15, 23 September 2016
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Overview
Research is underway into novel therapies to help in the management and possible cure of hemophilia A.
Gene therapy
The goal of gene therapy is to correct the chromosomal deficit which results in Hemophilia A. Currently, there is no role for gene therapy in the management of hemophilia A, though the issue is being pursued in clinical trials. [1] Researchers are also studying the genomes of patients affected with inhibitors to determine why some patients develop inhibitors and others do not. Understanding a patient's genetic profile may help physicians predict a patient's risk for developing inhibitors to factor concentrates, and help guide their treatment. [2]
Future and Investigational Therapies
References
- ↑ Konkle BA, Josephson NC, Nakaya Fletcher S. Hemophilia A. 2000 Sep 21 [Updated 2014 Jun 5]. In: Pagon RA, Adam MP, Ardinger HH, et al., editors. GeneReviews® [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2016. Available from: http://www-ncbi-nlm-nih-gov.laneproxy.stanford.edu/books/NBK1404/
- ↑ Why We Do research on Hemophilia | Hemophilia | NCBDDD | CDC. Available at http://www.cdc.gov/ncbddd/hemophilia/research.html. Accessed on Sept 20, 2016