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Medication for Rare Diseases

Disease

Medication

urea cycle disorder

Arginine HCL Injection

Homocystinuria

Cystadane

Congenital hyperammonemia caused by abnormal urea metabolism due to citrulline deficiency

Stimol

Acute and chronic treatment of secondary carnitine deficiency patients with congenital genetic abnormalities of metabolism

L-Carnitine

urea cycle disorder

Buphenyl

Tetrahydrobiopterin dificiency

BH4

The hyperinsulinism/hyperammonemia syndrome

Proglycem (Diazoside)

Non-Ketotic hyperglycinemia

Sodium Benzoate

Porphyria

Normosang

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