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
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