Etranacogene dezaparvovec (AMT-061 phase 2b): normal/near normal FIX activity and bleed cessation in hemophilia B.
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DOI: 10.1182/bloodadvances.2019000811
Abstract: Etranacogene dezaparvovec (AMT-061) is a recombinant AAV5 vector including a gene cassette containing the factor IX (FIX) Padua variant under the control of a liver-specific promoter. A phase 2b study was conducted to confirm that… read more here.
Keywords: activity; cessation; dezaparvovec amt; fix activity ... See more keywords