Hyperphenylalaninemia and treatments thereof
Abstract:
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.
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