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Article

Etranacogene dezaparvovec for hemophilia B gene therapy

1 Jan 2021

Abstract excerpt

The treatment landscape for hemophilia has been rapidly changing with introduction of novel therapies. Gene therapy for hemophilia is a promising therapeutic option for sustained endogenous factor production to mitigate the need for prophylactic treatment to prevent spontaneous and traumatic bleeding. Etranacogene dezaparvovec is an investigational factor IX (FIX) gene transfer product that utilizes the...

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PMID: 37181105
DOI: 10.1177/26330040211058896

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