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When the phosphatidylinositol glycan anchor biosynthesis Class A (<jats:italic>PIGA<\/jats:italic>) gene was identified by Taroh Kinoshita\u2019s group, jointly with him the Hammersmith group proved that PNH is caused in most patients by a single somatic mutation in the <jats:italic>PIGA<\/jats:italic> gene. At the same time, after Bruno Rotoli had spent a sabbatical at the Hammersmith, the \u2018immune escape model\u2019 for the pathogenesis of PNH was developed. Early this century, Peter Hillmen, formerly at the Hammersmith and now in Leeds, spearheaded the use of the complement\u2010blocking (anti\u2010C5) antibody eculizumab. This new medicine radically changed the management and the clinical course of patients with PNH. Recently a derivative of eculizumab with more favourable pharmacokinetics has been introduced. In view of the fact that these agents are associated with C3\u2010dependent extravascular haemolysis, it is important that a number of inhibitors of the proximal complement pathway are now in the offing and may further improve the life of patients with PNH.<\/jats:p>","DOI":"10.1111\/bjh.17147","type":"journal-article","created":{"date-parts":[[2023,9,5]],"date-time":"2023-09-05T01:19:19Z","timestamp":1693876759000},"page":"579-586","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":9,"title":["Paroxysmal nocturnal haemoglobinuria (PNH): novel therapies for an ancient disease"],"prefix":"10.1111","volume":"191","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4663-8790","authenticated-orcid":false,"given":"Lucio","family":"Luzzatto","sequence":"first","affiliation":[{"name":"Department of Haematology and Blood Transfusion Muhimbili University of Health and Allied Sciences Dar\u2010es\u2010Salaam Tanzania"},{"name":"University 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