Margareth C. Ozelo

Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial

Two-Year Outcomes of Valoctocogene Roxaparvovec Therapy for Hemophilia A

Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe hemophilia A in the phase 3 trial GENEr8-1

Global Seroprevalence of Pre-existing Immunity Against AAV5 and Other AAV Serotypes in People with Hemophilia A

International consensus recommendations on the management of people with haemophilia B

First hemophilia B gene therapy approved: More than two decades in the making

ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease

von Willebrand disease: proposing definitions for future research.

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Long‐term safety and sustained efficacy for up to 5 years of treatment with recombinant factor IX Fc fusion protein in subjects with haemophilia B: Results from the B‐YOND extension study

Long-term safety and efficacy of extended-interval prophylaxis with recombinant factor IX Fc fusion protein (rFIXFc) in subjects with haemophilia B

Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

Results from a large multinational clinical trial (guardian™1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia <scp>A</scp>: safety and efficacy