A review of the treatment landscape in paroxysmal nocturnal haemoglobinuria: where are we now and where are we going?
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Paroxysmal nocturnal haemoglobinuria (PNH) is an ultra-orphan disease, which until 15 years ago had limited treatment options. Eculizumab, a monoclonal antibody that inhibits C5 in the terminal complement cascade, has revolutionised treatment for this disease,... ...
