PNH
Clinical trials for PNH explained in plain language.
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New hope for kids with rare blood disease: Add-On pill may boost red blood cells
Disease control Recruiting nowThis study tests whether adding danicopan (a pill) to standard PNH therapy (ravulizumab or eculizumab) can improve hemoglobin levels in children aged 12 to 17 with paroxysmal nocturnal hemoglobinuria (PNH) who still have anemia due to extravascular hemolysis. About 6 participants…
Matched conditions: PNH
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 17, 2026 05:05 UTC
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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 anemia despite current treatment with Soliris or Ultomiris. Participants must be at least 18 years old, have been on Soliris or Ultomiris for 6 months, a…
Matched conditions: PNH
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 11, 2026 20:38 UTC