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.
本发明涉及说明书中定义的式(I)化合物,用作对苯丙
氨酸羟化酶(PAH)具有稳定作用的药理合剂,用于治疗高苯丙
氨酸血症(HPA),特别是
苯丙酮尿症(PKU)。这些化合物包括三
甲氧苄啶、其类似物和衍
生物。