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The impact of capping health system cost savings on the projected cost-effectiveness of etranacogene dezaparvovec compared with factor IX prophylaxis for the treatment of hemophilia B. [PDF]
J Manag Care Spec PharmSarker J +4 more
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Seminars in Thrombosis and Hemostasis, 2018
Hemophilia B (HB) is an X-linked bleeding disorder caused by deficiency of factor IX (FIX). Patients with the severe form (FIX
C. Santoro +6 more
semanticscholar +4 more sources
Hemophilia B (HB) is an X-linked bleeding disorder caused by deficiency of factor IX (FIX). Patients with the severe form (FIX
C. Santoro +6 more
semanticscholar +4 more sources
Current factor IX replacement options for hemophilia B and the challenges ahead
Expert Opinion on Pharmacotherapy, 2023Introduction Therapy for hemophilia B is aimed at replacing the congenital deficiency of coagulation factor IX (FIX). For replacement therapy, several FIX concentrates derived from donated human plasma or engineered by recombinant DNA technology are ...
M. Franchini, M. Zaffanello, D. Focosi
semanticscholar +1 more source
Hematology/Oncology Clinics of North America, 2022
Adeno-associated virus (AAV)-mediated gene transfer has successfully raised, and in some cases transiently normalized, FVIII or FIX activity levels in adults with severe hemophilia. Raising FVIII/IX levels, particularly greater than ∼15 IU/dL (mild deficiency), corresponds to a marked decrease in spontaneous and provoked bleeding, dramatic reduction in
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Adeno-associated virus (AAV)-mediated gene transfer has successfully raised, and in some cases transiently normalized, FVIII or FIX activity levels in adults with severe hemophilia. Raising FVIII/IX levels, particularly greater than ∼15 IU/dL (mild deficiency), corresponds to a marked decrease in spontaneous and provoked bleeding, dramatic reduction in
openaire +2 more sources
Acta Haematologica, 2001
Hemophilia B is rare in females and only a few cases have been reported. In this report, we describe a girl with a clinically severe course of hemophilia B but with a normal 46,XX karyotype. She had no signs of Turner’s syndrome or any other dysmorphic features.
Shrimati Shetty +2 more
openaire +3 more sources
Hemophilia B is rare in females and only a few cases have been reported. In this report, we describe a girl with a clinically severe course of hemophilia B but with a normal 46,XX karyotype. She had no signs of Turner’s syndrome or any other dysmorphic features.
Shrimati Shetty +2 more
openaire +3 more sources