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...

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Bibliographic Details
Main Authors KAKKIS, Emil, D, PRICE, Fredric, D, DORENBAUM, Alejandro, OPPENHEIMER, Daniel I
Format Patent
LanguageEnglish
Hungarian
Published 28.03.2017
Subjects
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
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Summary: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.
Bibliography:Application Number: HUE04819152