PNH
Clinical trials for PNH explained in plain language.
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New hope for PNH patients: early access to danicopan combo therapy
Disease control AVAILABLEThis program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who have significant extravascular hemolysis (EVH) despite treatment with Soliris or Ultomiris. Participants must be 18 or older, have anemia and high reticulocyte counts, an…
Matched conditions: PNH
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 13:03 UTC
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New drug combo aims to boost red blood cells in kids with rare blood disorder
Disease control Recruiting nowThis phase 3 study tests whether adding danicopan to standard PNH medications (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria who still have significant anemia from extravascular hemolysis. Six participa…
Matched conditions: PNH
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC