Valoctocogene roxaparvovec gene therapy provides durable haemostatic control for up to 7 years for haemophilia A.

Emily SymingtonSavita RangarajanWilliam A LesterBella MadanGlenn F PiercePriyanka RahejaCarolyn MillarDane OsmondMingjin LiTara M Robinson

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2024)

The safety and efficacy of valoctocogene roxaparvovec remain generally consistent with previous reports, with good haemostatic control for most participants. Two participants returned to prophylaxis.

Keyphrases

gene therapy
emergency department
adverse drug