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The All Party Parliamentary Group (APPG) on Rare, Genetic and Undiagnosed Conditions published a report yesterday looking at the implementation of the UK Strategy for Rare Diseases in England. The APPG has found that the Strategy is not being effectively implemented in England. England is the only country in the United Kingdom where an implementation

In this issue of Blood, Harder et al demonstrate that breakthrough hemolysis in patients with paroxysmal nocturnal hemoglobinuria (PNH) treated with eculizumab is due to strong activation of the complement cascade, leading to higher C3b density, which may cause a conformation change limiting the ability of eculizumab to block terminal complement. Read More