Methods and compositions for the treatment of metabolic disorders
AssigneeBiomarin Pharmaceutical Inc.
InventorsDaniel I. Oppenheimer, Emil D. Kakkis, Fredric D. Price, Alejandro Dorenbaum, Rudolf Moser, Viola Groehn, Thomas Egger, Fritz Blatter
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Abstract
The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes methods and compositions for treating various types of phenylketonurias using compositions comprising BH4. Combination therapies of BH4 and other therapeutic regimens are contemplated.
Claims12
A method for treating a subject suffering from hyperphenylalaninemia (HPA) due to BH4-responsive phenylketonuria (PKU) comprising administering to said subject tetrahydrobiopterin (BH4) or pharmaceutically acceptable salt thereof at a daily dose of 10 mg/kg to 20 mg/kg, wherein the administering is multiday, oral, and only once per day.
Dependencies are inferred from the claim text.