Today, potential immunogenicity can be better evaluated during the drug development
process, and we have rational approaches to manage the clinical consequences of …

Although significant advances in hemophilia treatment have improved patient outcomes and
quality of life, one of the greatest complications in severe hemophilia A is the development of …

Hemophilia is a hereditary disease that remains incurable. Although innovative treatments
such as gene therapy or bispecific antibody therapy have been introduced, substantial …

Hemophilia A is an X-linked genetic disorder caused by mutations in the F8 gene, which
encodes the blood coagulation factor VIII. Almost half of all severe hemophilia A cases result …

The primary cellular source of factor VIII (FVIII) biosynthesis is controversial, with
contradictory evidence supporting an endothelial or hepatocyte origin. LMAN1 is a cargo …

The pathogenesis of inhibitory antibodies has been the focus of major scientific interest over
the last decades, and several studies on underlying immune mechanisms and risk factors for …

Phenotypic assays are first-line in terms of diagnostic testing for inherited bleeding
disorders. However, since the characterization of the genes that encode coagulation factors …

The development of inhibitors, ie neutralizing alloantibodies against factor (F) VIII or FIX, is
the most significant complication of protein replacement therapy for patients with hemophilia …

Nearly half of severe Hemophilia A (HA) cases are caused by F8 intron 22 inversion (Inv22).
This 0.6-Mb inversion splits the 186-kb F8 into two parts with opposite transcription …

Background Several studies have suggested Black and Hispanic hemophilia A (HA) patients
in the United States suffer higher incidences of neutralizing anti-FVIII antibodies (inhibitors) …