Register Login

Coagulation Factors

Acquired Hemophilia, rpVIII, and Bethesda Titer

Here is a 12-2-23 email from Hrvoje Melinšćak MD, Consultant in Haematology, the Warren Alpert Medical School of Brown University, Rhode Island Hospital/The Miriam Hospital. I would enquire regarding rpFVIII’s impact on inhibitor titre assessment through the Bethesda assay in a case regarding acquired haemophilia A. Does significant background rpFVIII (Obizur) concentrate lend to significant […]
read more

Follow-up to “FDA Approves Hemophilia A Gene Transfer Therapy”

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 […]
read more

FDA Approves Hemophilia A Gene Transfer Rx

Today, July 3, 2023, the FDA approved the use of BioMarin’s Roctavian® [valoctocogene roxaparvovec] for treating adults with severe hemophilia A without inhibitors. The FDA also approved BioMarin’s companion diagnostic, AAV5 DetectCDx, which identifies the patients most likely to benefit from Roctavian. Click here for the LinkedIn announcement. Click here for a provocative FirstWord PHARMA […]
read more