申请人:Universidad de Zaragoza
公开号:US08338423B2
公开(公告)日:2012-12-25
The present invention relates to the use of specific types of compounds acting as pharmacological chaperones having a stabilizing activity on the phenylalanine hydroxylase (PAH) enzyme for the prophylaxis and/or treatment of hyperphenylalaninemia (HPA) and phenylketonuria PKU) and diseases, disorders and conditions related thereto. Further, the present invention relates to pharmaceutical compositions containing the compounds according to the present invention as well as to methods of treatment of HPA, in particular, PKU and diseases, disorders and conditions related thereto.
本发明涉及使用特定类型的化合物作为药理伴侣子,在苯丙氨酸羟化酶(PAH)酶上具有稳定活性,用于预防和/或治疗高苯丙氨酸血症(HPA)和苯丙酮尿症(PKU)及相关疾病、疾病和状况。此外,本发明还涉及含有根据本发明的化合物的制药组合物,以及用于治疗HPA,特别是PKU和相关疾病、疾病和状况的治疗方法。