Clinical trial demonstrates sustained factor IX expression and low bleeding rates in hemophilia B, indicating potential long-term benefits.
Sustained endogenous factor IX expression and low annualized bleeding rates over a 5-year period were observed after an infusion of etranacogene dezaparvovec. (Funded by uniQure and CSL Behring; HOPE-B ClinicalTrials.gov number, NCT03569891.).
No takes yet. Share an insight, caveat, or question.
Miesbach et al. (2025) studied this question.
Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context: