Current factor IX replacement options for hemophilia B and the challenges ahead [PDF]
Expert Opinion on Pharmacotherapy, 2023 Introduction: 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 ...
Daniele Focosi +2 more
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A Case of Acquired Hemophilia A and Congenital Hemophilia B. [PDF]
Cureus, 2022 Congenital hemophilia B is a rare, inherited X-linked bleeding disorder caused by a deficiency of factor IX (FIX). Acquired hemophilia A is a rare, acquired bleeding disorder which presents as new onset bleeding in older adults due to the development of autoantibodies against factor VIII (FVIII).
Fortier JC +4 more
europepmc +2 more sources
A Post-Authorization Safety Surveillance Study to Report Clinical Experience with Purified Factor IX Concentrate in Pediatric Patients with Hemophilia B. [PDF]
J Blood MedIntroduction: Purified factor IX (FIX) concentrate (IMMUNINE®, Takeda Manufacturing Austria AG, Vienna, Austria) is indicated for the treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia B. Data on the use of purified FIX
Igrutinović Z +6 more
europepmc +3 more sources
Neurosurgery in a patient with severe hemophilia B: an experience using eftrenonacog alfa as perioperative management [PDF]
, 2022 Anesthesia; Haemophilia B; NeurosurgeryAnestesia; Hemofilia B; NeurocirugíaAnestèsia; Hemofília B; NeurocirurgiaExtended half-life FIX (EHL-FIX) concentrates have been developed with the purpose of reducing the frequency of infusions in patients with ...
Benitez Hidalgo, Olga +5 more
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Inhibitors in Patients with Congenital Bleeding Disorders Other Than Hemophilia [PDF]
, 2017 The most worrying complication of replacement therapy for severe hemophilia A and B is currently the occurrence of inhibitory alloantibodies against infused factor VIII and factor IX, respectively.
Franchini, Massimo +6 more
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Haemophilia and joint disease: pathophysiology, evaluation and management [PDF]
, 2011 In patients with haemophilia, regular replacement therapy with clotting factor concentrates (prophylaxis) is effective in preventing recurrent bleeding episodes into joints and muscles.
Berntorp, Erik, Knobe, Karin
core +3 more sources
In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A. [PDF]
, 2018 Hemophilia A (HA) is an X-linked recessive disorder caused by mutations in the factor VIII ( FVIII) gene leading to deficient blood coagulation. The current standard of care is frequent infusions of plasma-derived FVIII or recombinant B-domain-deleted ...
Farmer, Diana +6 more
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Complete Genotype and Clinical Phenotype of Hemophilia B: A Study on Iranian Patients [PDF]
, 2021 Background: Hemophilia B which refers to the deficiency or functional defect of factor IX (FIX) is typically an X-linked bleeding condition that arises from heterogeneous mutations of the FIX gene (F9).
Dorgalaleh, Akbar +4 more
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Recombinant factorVIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A [PDF]
, 2015 This work was supported by funding from Biogen, including funding for the editorial and writing support in the the development of this ...
Allen, G +11 more
core +1 more source
Chemical Chaperones Improve Protein Secretion and Rescue Mutant Factor VIII in Mice with Hemophilia A. [PDF]
, 2012 nefficient intracellular protein trafficking is a critical issue in the pathogenesis of a variety of diseases and in recombinant protein production.
Abriss, Daniela +9 more
core +2 more sources