AV513 is a select fucoidan, a sulfated polysaccharide of botanical origin. It inhibits tissue factor pathway inhibitor (TFPI) activity and accelerates clotting of human hemophilia A and B plasma. In prior work, subcutaneous administration of AV513 to mice with hemophilia A improved hemostasis. The current studies were designed to evaluate potential efficacy and safety in dogs with hemophilia A ...

The majority of cases of human hemophilia B are the result of missense mutations in the coagulation factor IX gene and defective circulating factor IX is detectable in most patients. The available mouse factor IX knockout models of hemophilia B (FIXKO mouse) reproduce the bleeding phenotype of human hemophilia B, but because the models produce no factor IX they fail to reproduce the dominant hu...

Preclinical testing of new therapeutic strategies in relevant animal models is an essential part of drug development. The choice of animal models of disease that are used in these studies is driven by the strength of the translational data for informing about safety, efficacy, and success or failure of human clinical trials. Hemophilia B is a monogenic, X-linked, inherited bleeding disorder tha...

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, including a recently completed phase I/II, open-label, multicenter, study that assessed the safety, phar...

Hemophilia B, an X-linked recessive bleeding disorder is caused by deficiency of clotting factor 1X protein. In an attempt to understand the molecular basis of hemophilia B which is a rare disease in Pakistan, we analyzed amplified genomic DNA from 20 patients (ages 3-36 years) with different ethnic back-grounds. Eleven mutations were identified in 15 out of 20 patients with mutation detection ...

TUI)IES OF HEMOPHILIA have, ins the past-, helped greatly to solve the mansy intricacies of the blood coagulation mechanism. More recently, howeser, they have also altered our s-cry (‘onscept of the disease arid stnggested that, ins-stead of a svehl defined entity, hemophilia ins fact represents a syndrome siit.h many different pathogensetic mechanisms. The questions “ i’hat. is hemophihia?” is...

background hemophilia might impact the quality of life (qol) in children and adolescent. this study aimed to assess the quality of life in children with hemophilia and identify the factors that predict their qol. methods it was a cross-sectional study. a consensus sample of twenty-seven male children aged 8-16 years old with hemophilia participated in this study during 2011. the haemo-qol quest...

Activated factor VII is approved for treating hemophilia patients with autoantibodies to their factor IX or FVIII; however, its mechanism of action remains controversial. Some studies suggest that FVIIa requires tissue factor (TF) for function and that the reason for the high dose requirement is that it must compete with endogenous FVII for tissue factor. Others suggest that FVIIa binds platele...

Hemophilia A and B are caused by deficiencies in coagulation factor VIII (FVIII) and factor IX, respectively, resulting in deficient blood coagulation via the intrinsic pathway. The extrinsic coagulation pathway, mediated by factor VIIa and tissue factor (TF), remains intact but is negatively regulated by tissue factor pathway inhibitor (TFPI), which inhibits both factor VIIa and its product, f...