Evaluation of One‐Stage Assays for the Monitoring of Recombinant Human Factor IX Padua Activity After Etranacogene Dezaparvovec Gene Therapy
FIX activity differences between central and local OSAs were modest; similar differences were observed in vitro with rhFIX-wt versus rhFIX-Padua. Commonly available OSAs can be used to monitor patients post-etranacogene dezaparvovec administration; we recommend using the same assay platform throughout the post-treatment period.
Authors
- Michiel Coppens (ORCID: https://orcid.org/0000-0001-6891-9062)
- Guy A. Young (ORCID: https://orcid.org/0000-0001-6013-1254)
- Nick Galante
- Jan Astermark (ORCID: https://orcid.org/0000-0001-8500-2483)
- Jaap Twisk
- Paul E. Monahan (ORCID: https://orcid.org/0000-0003-2110-0740)
- Ricardo Dolmetsch
- Stephanie Verweij
- Sander Gielen
- Bruce M. Ewenstein (ORCID: https://orcid.org/0000-0003-3871-8619)
- Silpa Nuthalapati (ORCID: https://orcid.org/0000-0001-5220-2868)
- Wolfgang Miesbach (ORCID: https://orcid.org/0000-0002-4506-0061)
Institutions
- Goethe University Frankfurt (DE)
- University of Southern California (US)
- Lund University (SE)
- CSL (United States) (US)
- UniQure (Netherlands) (NL)
- University Hospital Frankfurt (DE)
- Skåne University Hospital (SE)
- Amsterdam University Medical Centers (NL)
- University of Amsterdam (NL)
Publication Details
- Journal
- Haemophilia
- Published
- 2025-06-18
- DOI
- https://doi.org/10.1111/hae.70053
- Citations
- 2
- Primary Topic
- Hemophilia Treatment and Research
- Type
- article
- Field-Weighted Citation Impact
- 3.54
Funders
- CSL Behring