New hope for PNH patients: early access to additional treatment

NCT ID NCT05982938

Summary

This 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 and have specific blood test results showing ongoing anemia. The program is for patients who cannot participate in clinical trials and whose doctors believe the potential benefits outweigh the risks.

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