That is the question now being considered in light of a new research article in the New England Journal of Medicine in which a new gene therapy drug was given to men with hemophilia B. After receiving the new drug, the patients were able to stop using clotting factors to treat their hemophilia. Patients rates of bleeding also fell - from about 11 bleeding events per year to less than one bleeding event per year. Learn more about the research here:
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- van den Berg, H M. (2017). A cure for hemophilia within reach. The New England Journal of Medicine, doi:10.1056/NEJMe1713888 [doi]. Epub ahead of print.
- Porteus, M. (2017). Closing in on treatment for hemophilia B. The New England Journal of Medicine, 377(23), 2274-2275. doi:10.1056/NEJMe1713735 [doi]
- George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., . . . High, K. A. (2017). Hemophilia B gene therapy with a high-specific-activity factor IX variant. The New England Journal of Medicine, 377(23), 2215-2227. doi:10.1056/NEJMoa1708538 [doi]
[youtube https://www.youtube.com/watch?v=6TpLoZCaL3c]
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