Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS) research hub

A Phase 3, Open-Label, Single-arm, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Activity, and Safety of Ravulizumab Administered Subcutaneously in Pediatric Participants (2 to < 18 years of age) with Paroxysmal Nocturnal Hemoglobinuria (PNH) or Atypical Hemolytic Uremic Syndrome (aHUS)

This study looks at a new way to give medicine called ravulizumab (Ultomiris) to children aged 2 to 18 with two rare blood conditions, PNH or aHUS. We want to see how the medicine works, how safe it is, and if it helps them feel better.