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 Study. Ozelo MC, Luciani M, Glosli H, Kavakli K, Samji N, Makris GC, Tueckmantel C, Maas Enriquez M, Oliveira LC, Gupta S, Arbesú MG, Davoli M, Chan AKC, Mancuso ME.Eur J Haematol. 2026 Apr;116(4):435-442. DOI: 10.1111/ejh.70059
- 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 Study. Ozelo MC, Luciani M, Glosli H, Kavakli K, Samji N, Makris GC, Tueckmantel C, Enriquez MM, Oliveira LC, Gupta S, Arbesú MG, Davoli M, Chan AKC, Mancuso ME.Eur J Haematol. 2026 Apr;116(4):471-472. DOI: 10.1111/ejh.70078
- Long-term durability of rAAV gene therapy in hemophilia: Factor expression, clinical outcomes and underlying molecular mechanisms. Dargaud Y, Yamaguti-Hayakawa GG, Frade-Guanaes JO, Leuci A, Ozelo MC. Blood Rev. 2026 Jan;75:101353. DOI: 10.1016/j.blre.2025.101353
2025
- Safety and efficacy of valoctocogene roxaparvovec with prophylactic glucocorticoids: 1-year results from the phase 3b, single-arm, open-label GENEr8-3 study. Ozelo MC, Manson J, Dunn AL, Villaça PR, Shen MC, Agarwal S, Imtiaz U, Liu H, Robinson TM. J Thromb Haemost. 2025 May;23(5):1496-1506. DOI: 10.1016/j.jtha.2024.12.03
- New insights into Wiskott-Aldrich syndrome: ten novel WAS mutations and their clinical impact in a Brazilian cohort. Santos LW, Medina SS, Frade-Guanaes JO, Siqueira LH, de Lima LGR, Chati B, Nolasco da Silva MT, Riccetto AGL, Lyra P, Falcão ACAM, Santos PPA, Di Gesu RSW, Stefanello B, Yamaguti-Hayakawa GG, Bonfim CMS, Vilela MMS, Ozelo MC.Front Immunol. 2025 Jul 31;16:1585594. DOI: 10.3389/fimmu.2025.1585594
- Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance. Konkle BA, Peyvandi F, Foster GR, Hermans C, La Mura V, Leavitt AD, Lillicrap D, Mahlangu J, Ozelo MC, Pipe S, Recht M, Srivastava A, Young G, Miesbach W.J Thromb Haemost. 2025 Mar 14:S1538-7836(25)00150-3.
DOI: 10.1016/j.jtha.2025.02.042 - Efficacy, safety, and quality of life 4 years after valoctocogene roxaparvovec gene transfer for severe hemophilia A in the phase 3 GENEr8-1 trial. Leavitt AD, Mahlangu J, Raheja P, Symington E, Quon DV, Giermasz A, López Fernández MF, Kenet G, Lowe G, Key NS, Millar CM, Pipe SW, Madan B, Chou SC, Klamroth R, Mason J, Chambost H, Peyvandi F, Majerus E, Pepperell D, Rivat C, Yu H, Robinson TM, Ozelo MC. Res Pract Thromb Haemost. 2024 Oct 30;8(8):102615.
DOI: 10.1016/j.rpth.2024.102615