| 摘要 |
The present invention relates to the use of specific types of compounds acting as pharmacological chaperones having a stabilizing activity op 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. |
| 申请人 |
UNIVERSIDAD DE ZARAGOZA;PEY RODRIGUEZ, ANGEL;MARTINEZ RUIZ, AURORA;SANCHO SANZ, JAVIER;CREMADES CASASIN, NUNILO;VELAZQUEZ CAMPOY, ADRIAN;YING, MING |
发明人 |
PEY RODRIGUEZ, ANGEL;MARTINEZ RUIZ, AURORA;SANCHO SANZ, JAVIER;CREMADES CASASIN, NUNILO;VELAZQUEZ CAMPOY, ADRIAN;YING, MING |
