Confirmation of longer FIX activity half-life with prolonged sample collection after single doses of nonacog alfa in patients with haemophilia B.
Sign Up to like & getrecommendations! Published in 2017 at "Thrombosis and haemostasis"
DOI: 10.1160/th16-10-0765
Abstract: A multicentre, single-dose study enrolled 12 previously treated patients with moderately severe to severe (factor IX [FIX] levels ≤2 IU/dl) haemophilia B to assess FIX pharmacokinetics after nonacog alfa administration and to evaluate the impact… read more here.
Keywords: sample collection; half life; life; fix activity ... See more keywords
A Single Intravenous Infusion of FLT180a Results in Factor IX Activity Levels of More Than 40% and Has the Potential to Provide a Functional Cure for Patients with Haemophilia B
Sign Up to like & getrecommendations! Published in 2018 at "Blood"
DOI: 10.1182/blood-2018-99-118050
Abstract: Introduction: Haemophilia B is an X-linked disease caused by mutations of the F9 gene coding for Factor IX (FIX). It is classified as mild, moderate, or severe based on FIX level, which correlates to clinical… read more here.
Keywords: week; infusion; fix; flt180a ... See more keywords
Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.
Sign Up to like & getrecommendations! Published in 2019 at "Blood advances"
DOI: 10.1182/bloodadvances.2019000811
Abstract: Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter. A phase 2b study was conducted to confirm that… read more here.
Keywords: activity; cessation; dezaparvovec amt; fix activity ... See more keywords