作者: Alejandro Dorenbaum , Emil D. Kakkis , Daniel I. Oppenheimer , Fredric D. Price , Thomas Egger
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摘要: The present invention is directed to a novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, specification describes treating various types of phenylketonurias using comprising BH4. Combination therapies BH4 other regimens are contemplated.