Neutralizing antibody formation against transgene products can represent a major complication following gene therapy with treatment of genetic diseases, such as hemophilia A. Although successful approaches have been developed to prevent the formation of anti-factor VIII (FVIII) antibodies, innovative strategies to overcome pre-existing anti-FVIII immune responses in FVIII-primed subjects are st...

Additional therapeutic options are needed for patients with bleeding disorders such as hemophilia A, hemophilia B, severe von Willebrand disease, and other rare factor deficiencies. A novel approach to improve coagulation in such clotting disorders has been identified that, parodoxically, involves heparinlike sulfated polysaccharides. Select molecules of this broad class are largely devoid of a...

Children with hemophilia (A or B) are at risk for bleeding episodes, which rank from mild mucosal/soft tissues bleeding to life-threatening hemorrhages. This report describes the dental/medical management provided to an 8.10-year-old patient suffering from uncontrolled bleeding after a surgical procedure to expose both permanent upper central incisors, in which hemophilia was a pure incidental ...

BACKGROUND Hemophilia is caused by deficiencies in coagulation factor VIII or IX, resulting in direct blockade of the intrinsic tenase complex and indirect blockade of the extrinsic tenase complex which is rapidly inhibited upon binding of factor Xa to tissue factor pathway inhibitor. We evaluated the ability of Gla-domainless factor Xa, a truncated form of factor Xa devoid of procoagulant prop...

Hemophilia B is an X-Iinked recessive bleeding disorder resulting from a deficiency of the coagulation factor IX (FIX) protein activity. a vitamin K-dependent serine protease active in both the intrinsic and extrinsic coagulation systems. DNA analyses of the factor IX gene in two unrelated patients with severe hemophilia B. with a IX coagulant activity <1 % and undetectable FIX antigen. detecte...

The coagulation factors VII, IX, and X are all vitamin K dependent serine proteases synthesized in the liver with a high degree of similarity concerning size and structure. Factor IX is the deficient protein in hemophilia B and used in substitution therapy. The activated form of factor VII (FVIIa) is used to treat hemophilia in which the coagulant tenase complex cannot form due to inhibitory an...

BACKGROUND The main clinical manifestations of hemophilia are muscle and joint bleeding. Recurrent bleeding leads to a degenerative process known as hemophilic arthropathy. The development of inhibitors (antibodies against FVIII/FIX concentrates) is the main complication in the treatment of hemophilia. The objective was to assess the safety and efficacy of manual therapy treatment in a patient ...

Among bleeding disorders, hemophilia A, hemophilia B and von Willebrand diseases are the most commonly occurring, whereas deficiencies of other coagulation factors are rare worldwide. Unlike hemophilias, which are X-chromosome linked disorders, the inheritance pattern of the rare coagulation factor deficiencies is generally autosomal recessive. In literature, association of these factor deficie...

Background Hemophilia A and B are disorders resulting from a deficiency in factor VIII (FVIII), and factor IX (FIX), respectively. Tissue Factor (TF) is a key component of the extrinsic pathway and plays a role in the coagulation defect of hemophilic blood. Neutralizing the activity of TFPI represents a promising treatment concept in patients with hereditary or acquired hemophilia. ARC19499 is ...