PNH
Clinical trials for PNH explained in plain language.
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New hope for PNH patients: early access to additional treatment
Disease control AVAILABLEThis program provides early access to danicopan for adults with paroxysmal nocturnal hemoglobinuria (PNH) who are already receiving standard treatments but still experience significant red blood cell destruction. Participants must be on SOLIRIS or ULTOMIRIS for at least 6 months …
Matched conditions: PNH
Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Apr 02, 2026 05:11 UTC
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New hope for kids with rare blood disorder
Disease control Recruiting nowThis study is testing whether adding a new drug called danicopan helps children with a rare blood disease called PNH. The children are already on standard treatment but still have significant anemia. The main goal is to see if the add-on drug increases their hemoglobin levels (a …
Matched conditions: PNH
Phase: PHASE3 • Sponsor: Alexion Pharmaceuticals, Inc. • Aim: Disease control
Last updated Mar 27, 2026 12:38 UTC