Area of research
Hematology · Genetics · gene therapy AAV
Research interest
Research interests include Hemophilia Treatment and Research, Blood Coagulation and Thrombosis Mechanisms, Platelet Disorders and Treatments, and Virus-based gene therapy research.
The WFH Guidelines for the Management of Haemophilia: AAV Gene Therapy, 2025.
A comprehensive care pathway of gene therapy for hemophilia based on current guideline documents and summary of product characteristics: communication from the ISTH SSC working group on gene therapy.
Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years
Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study.
Accurate evaluation of factor VIII activity of efanesoctocog alfa in the presence of emicizumab
Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B.
Natural history of preexisting AAV5 antibodies in adults with hemophilia B during the lead-in of the etranacogene dezaparvovec phase 3 study
Completion of phase 2b trial of etranacogene dezaparvovec gene therapy in patients with hemophilia B over 5 years.
Development of a novel gene editing lexicon for hemophilia: methodology and results
Gene therapy for hemophilia B: results from the phase 1/2 101HEMB01/02 studies.
Extending health equity to people with moderate and mild hemophilia A: revisiting the HAVEN 6 trial
Global Comparative Antithrombin Field Study: Impact of Laboratory Assay Variability on the Assessment of Antithrombin Activity Measurement at Fitusiran Clinical Decision-Making Points.
Pain Reduction Following Eptacog Beta Treatment of Bleeding Episodes in Adolescents and Adults With Haemophilia A or B Complicated by Inhibitors.
Operationalising a Haemophilia Gene Editing Lexicon for Practical Use.
Treatment of severe bleeds with eptacog beta in hemophilia A or B with inhibitors: a post hoc analysis of the PERSEPT 1 and 2 trials
Guidelines for management of hemophilia—why, what, and how?
Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A
Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial.
Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial
Long-term outcomes with emicizumab in hemophilia A without inhibitors: results from the HAVEN 3 and 4 studies
A global comparative field study to evaluate the factor VIII activity of efanesoctocog alfa by one-stage clotting and chromogenic substrate assays at clinical haemostasis laboratories.
Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE-B trial 2 years after gene therapy.
A novel gene editing lexicon strategy for the haemophilia community: Research plan for development and preliminary results.
Transitioning from emicizumab prophylaxis to valoctocogene roxaparvovec gene therapy: A simulation study for individuals with severe haemophilia A.
Evaluation of an automated von Willebrand factor glycoprotein IbM activity assay compared with 3 alternative von Willebrand factor activity assays.
Experience with trametinib in a pediatric patient with MAP2K1-related cervicofacial arteriovenous malformation.
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A
Molecular evaluation and vector integration analysis of HCC complicating AAV gene therapy for hemophilia B.