HYPERPHENYLALANINEMIA AND TREATMENTS THEREOF

The present invention relates to compounds of formula (I) as defined in the specification for use as pharmacological chaperones having a stabilizing effect on phenylalanine hydroxylase (PAH) for the treatment of hyperphenylalaninemia (HPA), in particular phenylketonuria (PKU). The compounds include...

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Bibliographische Detailangaben
Hauptverfasser:	MARTINEZ, Aurora, CATEVILLA, Oscar Aubi, YING, Ming, UNDERHAUG, Jarl, SKJÆRVEN, Lars, TEIGEN, Knut
Format:	Patent
Sprache:	eng ; fre
Schlagworte:	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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Beschreibung
Zusammenfassung:	The present invention relates to compounds of formula (I) as defined in the specification for use as pharmacological chaperones having a stabilizing effect on phenylalanine hydroxylase (PAH) for the treatment of hyperphenylalaninemia (HPA), in particular phenylketonuria (PKU). The compounds include trimethoprim and analogues and derivatives thereof. La présente invention concerne des composés de formule (I) tels que définis dans la description destinés à être utilisés en tant que chaperons pharmacologiques présentant un effet stabilisant sur la phénylalanine hydroxylase (PAH) pour le traitement de l'hyperphénylalaninémie (HPA), en particulier de la phénylcétonurie (PKU). Les composés comprennent le triméthoprime et des analogues et dérivés de celui-ci.