Results 31 to 40 of about 13,000,271 (286)
Cochlear implantation in hemophilia B—a rare case report
The Egyptian Journal of Otolaryngology, 2022 Background Hemophilia B is an X-linked inherited disease, mainly caused by deficiency of factor IX. Severity of the disease is manifested by the factor IX deficiency in the blood.
Chetan Yadav +3 more
doaj +1 more source
Molecular Therapy: Methods & Clinical Development, 2017 Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX).
Shunsuke Iizuka +4 more
doaj +1 more source
Etranacogene dezaparvovec for hemophilia B gene therapy
Therapeutic Advances in Rare Disease, 2021 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 ...
C. Thornburg
semanticscholar +1 more source
Journal of Thrombosis and Haemostasis, 2020 An international, multicenter extension study evaluated recombinant fusion protein linking recombinant coagulation factor IX (FIX) with recombinant human albumin (rIX‐FP) in hemophilia B (FIX ≤ 2%) patients previously enrolled in a phase III study or who
M. Mancuso +7 more
semanticscholar +1 more source
Gene therapy with adeno-associated virus vector 5-human factor IX in adults with hemophilia B.
Blood, 2018 Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for FIX concentrate.
Wolfgang A. Miesbach +13 more
semanticscholar +1 more source
Immune tolerance induction in a severe hemophilia B child with low titer inhibitors
Journal of Applied Hematology, 2022 Hemophilia B is an X-linked inherited bleeding disorder caused by either the absence or reduced biosynthesis of clotting factor IX (FIX). This disorder affects approximately 1 in 30,000 male individuals worldwide. Patients with severe form (FIX
Asia Almulla, Najam Awan, Faisl Khanani
doaj +1 more source
Research and Practice in Thrombosis and Haemostasis, 2020 We report the first analysis of an extended half‐life recombinant factor IX, nonacog beta pegol (N9‐GP), in previously untreated patients (PUPs) and minimally treated patients with hemophilia B.
Anthony K. C. Chan +6 more
semanticscholar +1 more source
A Case of Heel Reconstruction with a Reverse Sural Artery Flap in a Hemophilia B Patient [PDF]
Archives of Plastic Surgery, 2012 Hemophilia B is a rare blood coagulation disorder. Complications such as bleeding and hematoma can cause necrosis of flaps, wound disruption, and the disturbance of wound healing.
Byung Kwon Lee, Jeong Su Shim
doaj +2 more sources
Blood Advances, 2019 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.
Annette von Drygalski +11 more
semanticscholar +1 more source
Development of a Clinical Candidate AAV3 Vector for Gene Therapy of Hemophilia B.
Human Gene Therapy, 2020 Although recombinant adeno-associated virus serotype 8 (AAV8) and serotype 5 (AAV5) vectors have shown efficacy in Phase 1 clinical trials for gene therapy of hemophilia B, it has become increasingly clear that these serotypes are not optimal for ...
Harrison C. Brown +7 more
semanticscholar +1 more source