TREATMENT OF PHENYLKETONURIA WITH BH4

The present invention is directed to novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes compositions comprising tetrahydrobiopterin (BH4) and the use thereof in methods of treating various types of phenylketonurias...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: OPPENHEIMER DANIEL I, KAKKIS EMIL D, DORENBAUM ALEJANDRO, PRICE FREDRIC D
Format: Patent
Sprache:eng ; pol
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Beschreibung
Zusammenfassung:The present invention is directed to novel methods and compositions for the therapeutic intervention in hyperphenylalaninemia. More specifically, the specification describes compositions comprising tetrahydrobiopterin (BH4) and the use thereof in methods of treating various types of phenylketonurias. Combination therapies of BH4 and other therapeutic regimens are contemplated.