Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment.
Emily SymingtonSavita RangarajanWilliam A LesterBella MadanGlenn F PiercePriyanka RahejaTara M RobinsonDane OsmondChris B RussellChristian VettermannSuresh K AgarwalMingjin LiWing Yen WongMichael A LaffanPublished in: Haemophilia : the official journal of the World Federation of Hemophilia (2024)
Valoctocogene roxaparvovec safety and efficacy profiles remain largely unchanged; genomic investigations showed no association with a parotid tumour.