Abstract
What is this summary about? Eculizumab and ravulizumab are approved treatments for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disease which can cause potentially fatal complications if left untreated. Long-term ravulizumab treatment in patients with PNH is under investigation in two ongoing studies (‘301’ and ‘302’). This article describes the results at 2 years for both studies. What were the results? Ravulizumab continued to manage most patients' symptoms and less than 3% of patients experienced serious side effects related to treatment during this time. What do the results mean? This article highlights why eculizumab and ravulizumab are the usual treatments for PNH, where available. Long-term, ravulizumab controlled patients' PNH disease activity with few side-effects related to treatment.
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