Gene therapy for people with hemophilia B: a proposed care delivery model in Italy

Gene therapy is designed to provide people with hemophilia B with a steady and elevated factor (F)IX activity, thereby strengthening protection and relieving the burden of frequent replacement therapy infusions. The European Medicines Agency has approved gene therapy for the severe and moderately se...

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Veröffentlicht in:	Journal of thrombosis and haemostasis 2024-11, Vol.22 (11), p.3084-3096
Hauptverfasser:	Castaman, Giancarlo, Di Minno, Giovanni, Simioni, Paolo, Molinari, Angelo Claudio, Siragusa, Sergio, Baldacci, Erminia, La Mura, Vincenzo, Lupi, Angelo, Grazzi, Enrico Ferri, Peyvandi, Flora
Format:	Artikel
Sprache:	eng
Schlagworte:	factor IX-Padua gene therapy hemophilia B organizational model patient care team patient outcome assessment
Online-Zugang:	Volltext
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