This is a follow-up to “FDA Approves Hemophilia A Gene Transfer Therapy,’ posted July 3, 2023, with reference to Dr. Emmanuel Favaloro’s comment, added August 9 and citing Valentino LA, Kaczmarek R, Pierce GF, et al. Hemophilia gene therapy: first, do no harm. J Thromb Haemost. 2023: S1538-7836(23)00501-9. doi: 10.1016/j.jtha.2023.06.016. [click for a copy]. The article carefully examines the efficacy and safety profiles of the recently approved gene transfer therapies for hemophilia A and B.
Aug 10 2023
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