Eculizumab is indicated for the treatment of patients with Paroxysmal Nocturnal Hemoglobinuria and atypical Hemolytic Uremic Syndrome
Rocket will also collaborate with investigators to implement an immunomodulatory regimen more closely reflecting that administered in the Phase 1 pediatric cohort
ALPHA Phase III trial showed first-in-class, oral, Factor D inhibitor as add-on to Ultomiris or Soliris improved haemoglobin levels and reduced anaemia and fatigue
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