Haemophilias A and B

Patient has a diagnosis or suspected diagnosis of hemophilia B and you would like to identify the underlying mutation Patient has a family history of hemophilia B Patient is a known or suspected carrier for hemophilia B, and the mutation in the family has not been previously identified

GUIDELINES FOR DENTAL TREATMENT OF PATIENTS WITH INHERITED

hemophilia B (Christmas disease) is a deficiency of factor IX Hemophilia is considered severe when plasma activity is

Hemophilia B Gene Therapy in Mice using a Novel Chimeric AAV

Hemophilia B Gene Therapy in Mice using a Novel Chimeric AAV Capsid Combined with a Potency Enhanced FIX Variant Gene Therapy for Blood Disorders March 3 rd - 5 th 2020 Grant E Blouse, PhD SVP Translational Research

Haemophilias A and B - The Lancet

Haemophilias A and B Paula H B Bolton-Maggs, K John Pasi The haemophilias are inherited disorders in which one of the coagulation factors is deficient Although deficiencies of factor VIII (haemophilia A) and factor IX (haemophilia B) are well recognised, von Willebrand’s disease is much more common

Review Article HEMOPHILIA - AN OVERVIEW

The occurrence of hemophilia –A is 1: 5000-10000 1, 2, 3, 4 Hemophilia B (Christmas disease) It is also an “X” linked recessive disorder occurring due to the absence or deficiency of the clotting factor IX The inheritance pattern and the symptoms of hemophilia B are same as that of the classic hemophilia The

Hemophilia: “The Royal Disease”

Hemophilia: “The Royal Disease” Hemophilia is an X-linked recessive disorder characterized by the inability to properly form blood clots Until recently, hemophilia was untreatable, and only a few hemophiliacs survived to reproductive age because any small cut or internal hemorrhaging after even a minor bruise were fatal

ITI with high-dose FIX and combined immunosuppressive therapy

Haemophilia B, inhibitor, immune tolerance induction, immunosuppression Summary Inhibitor development is a rare but serious event in hemophilia B patients Management is hampered by the frequent occurrence of al-lergic reactions to factor IX, low success rates of current inhibitor elimination protocols and the risk of development of nephrotic syn-