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Stable and durable factor IX levels in patients with hemophilia B over 3 years after etranacogene dezaparvovec gene therapy
Etranacogene dezaparvovec (AMT-061) is a recombinant adeno-associated virus serotype 5 (AAV5) vector containing a codon-optimized Padua variant human factor IX (FIX) transgene with a liver-specific promoter. Here, we report 3-year outcomes from a phase 2b, open-label, single-dose, single-arm, multic...
Autores principales: | von Drygalski, Annette, Gomez, Esteban, Giermasz, Adam, Castaman, Giancarlo, Key, Nigel S., Lattimore, Susan U., Leebeek, Frank W. G., Miesbach, Wolfgang A., Recht, Michael, Gut, Robert, Dolmetsch, Ricardo, Monahan, Paul E., Le Quellec, Sandra, Pipe, Steven W. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
The American Society of Hematology
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10539871/ https://www.ncbi.nlm.nih.gov/pubmed/36490302 http://dx.doi.org/10.1182/bloodadvances.2022008886 |
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