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