Wednesday, 21 November 2007

Treatment of genetic diseases: in the real world. 2. Hemophilia

Hemophilia is an inherited bleeding disorders that was record since the ancient time.
The male new born has to be circumscribed for religious purpose, the affected individuals can be bleed until dead leading to exception for families with history of bleeding in male.
The most famous family with hemophilia is the Queen victorian royal family; there are many male suffered from this kind of severe bleeding disorders.
Once upon a time, those affected individulas died in before 10 years olf from sever intracranila bleeding. Nowadays, we know that these patients are lacking of clotting factor in their blood. Two common types of disease lack from differnet factor: factor 8 in hemophilia A and factor 9 in hemophilia B. These factors are found in normal individual plasma and that can be collected, concentrate and given to patiens in need. from the advance of genetic engineering, we can synthesized factor 8 or 9 with recombinant technology to produce large amount of factors independently for blood donors anymore, but they are still expensive and restricted for use in the most severe one.
These are the list of saving life products for hemophiliac patients:
Fresh Frozen Plasma
Cryoprecipitated (factor 8 rich)
Cryoremoved plasma (factor 9 rich)
Factor 8 concentrate plasma
Factor 9 concentrate plasma

Factor 7 recombinant: Novoseven from Novo Nordisk (can be used in both types)
Factor 8 recombinant: Advate from baxter, Helixate from Behring
Factor 9 recombinant: BeneFIX from Wyeth
Anti-Inhibitor Coagulant Complex FEIBA from Baxter (can be used in both types)
In mild hemophilia A or hemophiliac patients undegoing to perform oral procedure: transamin (tranexamic acid) from Daiichi

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