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1. Dosage

Complement inhibitor and enzyme/protein replacement therapy is FDA-approved for conditions characterized by enzyme deficiencies such as1-21:

  • Adenosine deaminase (ADA) deficiency in severe combined immunodeficiency (SCID)
  • Atypical hemolytic uremic syndrome (aHUS)
  • Congenital sucrase isomaltase deficiency (CSID)
  • Fabry disease
  • Gaucher disease
  • Hereditary angioedema (HAE)
  • Hypophosphatasia
  • Mucopolysaccharidoses (MPS) 
    • Hurler and Hurler-Scheie forms of MPS I
    • Hunter syndrome (MPS II)
    • Morquio A syndrome (MPS IVA)
    • Maroteaux-Lamy syndrome (MPS VI)
  • o    Sly syndrome (MPS VII)
  • Myasthenia gravis
  • Paroxysmal nocturnal hemoglobinuria
  • Severe congenital protein C deficiency