Etranacogene dezaparvovec-drlb gene therapy for patients with hemophilia B (congenital factor IX deficiency).

Sekayan, Tro; Simmons, Dana H; von Drygalski, Annette

Sekayan, Tro; Simmons, Dana H; von Drygalski, Annette.

Affiliation

Sekayan T; Division of Hematology/Oncology, Department of Medicine, University of California, San Diego, San Diego, CA, USA.
Simmons DH; Capitol Scientific, Washington DC, USA.
von Drygalski A; Division of Hematology/Oncology, Department of Medicine, University of California, San Diego, San Diego, CA, USA.

Expert Opin Biol Ther ; 23(12): 1173-1184, 2023.

Article in En | MEDLINE | ID: mdl-37962325

Subject(s)

Hemophilia B; Adult; Humans; Hemophilia B/genetics; Hemophilia B/therapy; Factor IX/genetics; Factor IX/therapeutic use; Genetic Therapy/methods; Hemorrhage/prevention & control

Key words

Annual bleeding rate; Hemgenix; Padua variant; etranacogene dezaparvovec-drlb; factor IX (FIX); gene therapy; glucocorticoid therapy; hemophilia B

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Hemophilia B Limits: Adult / Humans Language: En Journal: Expert Opin Biol Ther Journal subject: BIOLOGIA / TERAPEUTICA Year: 2023 Document type: Article Affiliation country: United States Country of publication: United kingdom

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