Brand names: Sapropterin Dihydrochloride
12.1 Mechanism of Action Sapropterin dihydrochloride is a synthetic form of BH4, the cofactor for the enzyme phenylalanine hydroxylase (PAH). PAH hydroxylates Phe through an oxidative reaction to form tyrosine. In patients with PKU, PAH activity is absent or deficient. Treatment with BH4 can activate residual PAH enzyme activity, improve the normal oxidative metabolism of Phe, and decrease Phe levels in some patients.
Contraindications
10 documented side effects by frequency
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Dosage Forms
Tablet
Route
Oral