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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Hauptverfasser: OPPENHEIMER DANIEL I, KAKKIS EMIL D, DORENBAUM ALEJANDRO, PRICE FREDRIC D
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creator OPPENHEIMER DANIEL I
KAKKIS EMIL D
DORENBAUM ALEJANDRO
PRICE FREDRIC D
description 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.
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subjects CHEMISTRY
HETEROCYCLIC COMPOUNDS
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS
title TREATMENT OF PHENYLKETONURIA WITH BH4
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