Hemophilia gene therapy: first, do no harm.
Valentino LA, Kaczmarek R, Pierce GF, Noone D, O'Mahony B, Page D, Rotellini D, Skinner MW.
Valentino LA, et al.
J Thromb Haemost. 2023 Sep;21(9):2354-2361. doi: 10.1016/j.jtha.2023.06.016. Epub 2023 Jun 21.
J Thromb Haemost. 2023.
PMID: 37353081
Free article.
Review.
Here, we review data leading to the regulatory authorization of valoctocogene roxaparvovec, an adeno-associated virus 5 gene therapy, in Europe to treat hemophilia A and etranacogene dezaparvovec-drlb in the United States and Europe to treat hemophilia B. We also pr …
Here, we review data leading to the regulatory authorization of valoctocogene roxaparvovec, an adeno-associated virus 5 gene therapy, in Eur …