Long-term tolerability, immunogenicity and efficacy of Nuwiq® (human-cl rhFVIII) in children with severe haemophilia A.

A KlukowskaShaji K KumarV VdovinS KnaubJ BichlerM JansenI DzhunovaR J Liesner

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2018)

Long-term treatment with Nuwiq® for the prevention of bleeds in children with severe haemophilia A was well tolerated, effective and reduced spontaneous bleeding by up to 70% compared with GENA-03.

Keyphrases

young adults
endothelial cells
early onset
open label
randomized controlled trial
drug induced
induced pluripotent stem cells
study protocol