Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort

BACKGROUND: Fitusiran, an investigational small interfering RNA therapy, reduces antithrombin production to rebalance hemostasis in people with hemophilia A or B, with or without inhibitors. OBJECTIVES: To evaluate the safety and efficacy of fitusiran treatment for people with moderate/severe hemoph...

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Detalhes bibliográficos
Publicado no:J Thromb Haemost
Main Authors: Pasi, K. John, Lissitchkov, Toshko, Mamonov, Vasily, Mant, Tim, Timofeeva, Margarita, Bagot, Catherine, Chowdary, Pratima, Georgiev, Pencho, Gercheva‐Kyuchukova, Liana, Madigan, Kate, Van Nguyen, Huy, Yu, Qifeng, Mei, Baisong, Benson, Craig C., Ragni, Margaret V.
Formato: Artigo
Idioma:Inglês
Publicado em: John Wiley and Sons Inc. 2021
Assuntos:
HAEMOSTASIS
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC8251589/
https://ncbi.nlm.nih.gov/pubmed/33587824
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1111/jth.15270
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