• Gaucher Disease
• Familial Hypercholesterolemia
• Congenital Afibrinogenemia
• Beta-Thalassemia
• Sickle Cell Disease
• Von Willebrand Disease
• Paroxysmal Nocturnal Hemoglobinuria
• Advanced Primary Hyperoxaluria Type 1
• Progressive Familial Intrahepatic Cholestasis
• Wilson Disease