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19 avril 2023
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- handle: 10670/1.ufjook
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Haemophilia Hematophilia Classic hemophilia Factor VIII deficiency Hemophilia A Hemorrhagic diathesisCiter ce document
vroby, « First gene therapy to treat haemophilia B », ELSIBI, ID : 10670/1.ufjook
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EMA has recommended granting a conditional marketing authorisation in the European Union (EU) for Hemgenix (etranacogene dezaparvovec) for the treatment of severe and moderately severe haemophilia B in adults who do not have factor IX inhibitors (auto-antibodies produced by the immune system which make factor IX medicines less effective). Hemgenix is the first gene therapy to treat haemophilia B. It is delivered as a single infusion. Etranacogene dezaparvovec, the active substance in Hemgenix...