PNH results from an acquired mutation of the PIG-A gene resulting in sensitivity of red cells to undergo intravascular haemolysis with a number of life-threatening downstream effects such as anaemia, transfusion requirement and most importantly, thromboembolic phenomena. The introduction of anti-complement therapy into clinical practice has transformed the outlook of this disease with benefit to nearly all symptomatic patients.  The success of the original (and only licensed) monoclonal antibody therapy has resulted in a search for alternative forms of inhibiting complement and a significant number of molecules acting via different (and similar) mechanisms are currently under investigation. The interface between complement and a variety of rare diseases (such as Gaucher Disease) are now becoming evident and complement inhibition is already proving to be clinically useful in a variety of other diseases.
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