Enhanced Procoagulant Activity of Select Hemophilia B Causing Factor IX Variants with Emicizumab.

Kyumin LeeJulia Q ChauYani B Suber Anna R SternbergAllyson PishkoLindsey A GeorgeVijay BhojBhavya S Doshi Benjamin J Samelson-Jones

Published in: Blood (2024)

Emicizumab improves the procoagulant activity of select loss-of-function FIX variants with likely dysfunctional assembly of the intrinsic Xase complex that cause hemophilia B. FVIII-mimetics may represent an alternative non-factor therapy for select hemophilia B patients.

Keyphrases

copy number
ejection fraction
newly diagnosed
prognostic factors
gene expression
dna methylation