1. Dosage

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• 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
• Wolman disease (lysosomal acid lipase (LAL) deficiency)
• Mucopolysaccharidoses (MPS) 
  • Hurler and Hurler-Scheie forms of MPS I; 
  • Hunter syndrome (MPS II); 
  • Morquio A syndrome (MPS IVA);
  • Maroteaux-Lamy syndrome (MPS VI);
  • Sly syndrome (MPS VII)
• Myasthenia gravis
• Neuromyelitis optica spectrum disorder
• Paroxysmal nocturnal hemoglobinuria
• Pompe disease
• Severe congenital protein C deficiency