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Molecular Therapy : the Journal of the American Society of Gene Therapy|July 11, 2006
Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia BHaiyan Jiang, Glenn F Pierce, Margareth C Ozelo, et al.Blood|September 30, 2010
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapyJonathan D Finn, Margareth C Ozelo, Denise E Sabatino, et al.Cytotherapy|April 3, 2026
Futuristic global cell and gene therapy regulations based on a review of today's leading frameworksShahrukh K Hashmi, M Abdullah Jamil, Josu de la Fuente, et al.Journal of Thrombosis and Haemostasis : JTH|March 16, 2025
Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidanceBarbara A Konkle, Flora Peyvandi, Graham R Foster, et al.Therapeutic Advances in Hematology|April 8, 2022
International consensus recommendations on the management of people with haemophilia BDaniel P Hart, Davide Matino, Jan Astermark, et al.American Journal of Hematology|September 25, 2004
Genetic variability of platelet glycoprotein Ibalpha geneMargareth C Ozelo, Devanira S P Costa, Lucia H Siqueira, et al.Haemophilia : the Official Journal of the World Federation of Hemophilia|June 5, 2020
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 studyK John Pasi, Kathelijn Fischer, Margaret Ragni, et al.European Journal of Haematology|January 5, 2026
Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main StudyMargareth C Ozelo, Matteo Luciani, Heidi Glosli, et al.European Journal of Haematology|January 14, 2026
Plain Language Summary on Safety and Efficacy of Damoctocog Alfa Pegol in Previously Treated Children Aged 7 to < 12 Years With Severe Haemophilia A in the Phase 3, Open Label Alfa-PROTECT Main StudyMargareth C Ozelo, Matteo Luciani, Heidi Glosli, et al.Blood Advances|February 27, 2023
Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia BAmy D Shapiro, Roshni Kulkarni, Margaret V Ragni, et al.Pageof 7