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This was due to the manifestations of the disease as well as thefact that Haemophilia B is one of the most well characterised rare diseases whereproof of principle and proof of concept biomarkers (plasma Factor IX levels andrecombinant protein consumption) correlate very well with the disease outcome(need for prophylaxis as an indication of the severity of disease) [ 33]. A significant portion of the chapter will be devoted todiscuss the clinical role of these agents in the modern era

A significant portion of the chapter will be devoted todiscuss the clinical role of these agents in the modern era.

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