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hemophilia b/albumiini

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Recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) is a novel recombinant albumin fusion protein designed to extend the half-life of recombinant factor IX (rFIX), which is used in the management of hemophilia B. Clinical evaluation of rIX-FP in humans is underway,

Improved kinetics of rIX-FP, a recombinant fusion protein linking factor IX with albumin, in cynomolgus monkeys and hemophilia B dogs.

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BACKGROUND Prophylaxis of hemophilia B, at present, requires multiple infusions of human factor (F)IX concentrates per week. A FIX molecule with a prolonged half-life has the potential to greatly improve the convenience of, and adherence to, prophylaxis. OBJECTIVE The aim of our studies was to
A phase 3b extension study evaluated the long-term safety and efficacy of a recombinant fusion protein-linking coagulation factor IX (FIX) with albumin (rIX-FP) for the routine prophylaxis and on-demand treatment of bleeding in pediatric hemophilia B

Population pharmacokinetics of a new long-acting recombinant coagulation factor IX albumin fusion protein for patients with severe hemophilia B.

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Essentials The new recombinant factor IX (FIX) albumin fusion protein (rIX-FP) has a prolonged half-life. A population pharmacokinetic (PK) model was based on FIX activity levels of hemophilia B patients. The model was used to simulate different dosing scenarios of rIX-FP to help guide dosing. The

Coagulation Factor IX (Recombinant), Albumin Fusion Protein (Albutrepenonacog Alfa; Idelvion®): A Review of Its Use in Haemophilia B.

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Albutrepenonacog alfa (Idelvion®), a fusion protein that genetically fuses recombinant factor IX (rFIX) with recombinant human albumin (rAlbumin), is indicated in the treatment of haemophilia B. This narrative review discusses the pharmacological properties and clinical data related to the use of

Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patients.

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BACKGROUND rIX-FP is a coagulation factor IX (recombinant), albumin fusion protein with more than fivefold half-life prolongation over other standard factor IX (FIX) products available on the market. OBJECTIVE This prospective phase II, open-label study evaluated the safety and efficacy of rIX-FP
Recombinant fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP; Idelvion®(†)) is an innovative new treatment designed to extend the half-life of factor IX (FIX) and ease the burden of care for hemophilia B patients. The rIX-FP clinical development program -

Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients.

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A recombinant fusion protein linking coagulation factor IX (FIX) with human albumin (rIX-FP) has been developed to facilitate hemophilia B treatment by less frequent FIX dosing. This first-in-human dose-escalation trial in 25 previously treated subjects with hemophilia B (FIX ≤ 2 IU/dL) examined the

Long-acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial.

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A global phase 3 study evaluated the pharmacokinetics, efficacy, and safety of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 63 previously treated male patients (12-61 years) with severe hemophilia B (factor IX [FIX] activity ≤2%). The study included 2 groups:

CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice

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Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 loaded by vectors could induce high rates of specific site genome editing and correct disease-causing mutations. However, most monogenic genetic diseases such as hemophilia are caused by different mutations dispersed in one

Efficacy and safety of long-acting recombinant fusion protein linking factor IX with albumin in haemophilia B patients undergoing surgery.

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BACKGROUND Recombinant factor IX fusion protein (rIX-FP) has been developed to improve the pharmacokinetic (PK) profile of factor IX (FIX), allowing maintenance of desired FIX activity between injections at extended intervals, ultimately optimizing haemophilia B treatment. OBJECTIVE To determine the

Excessive breakthrough bleeding in haemophilia B patients on factor IX-albumin fusion protein prophylactic therapy: A single centre case series.

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In a previous study, we prepared a monoclonal antibody (MoAb) to coagulation factor IX (FIX), designated 65-10, which interfered with the activation of FIX by the activated factor XI/Ca(2+) and neutralized the prolonged ox brain prothrombin time of hemophilia B(M) [11,12]. The location of the

A new treatment strategy for hemophilia B: incorporation of factor IX into red cell ghosts.

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Although patients with severe hemophilia B have lifelong spontaneous hemorrhage and crippling hemarthroses, patients with 0.01--0.03 U/ml Factor IX activity have a milder disease state. The clinical condition of severely affected individuals could potentially be improved by prolonging the half-life

Change of antigenic and neutralizing specificity in substitutional epitope peptides of hemophilia B inhibitor.

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We have previously described the epitope mapping and functional neutralization of three factor IX inhibitors in hemophilia B (HB-1, 3, and 7) by synthetic peptides (13). However, A concentration of synthetic peptide of about 1000 times the concentration of factor IX in plasma was essential to
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