Publicações Recentes LIMC

  • 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
  • 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.