A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/42 (2006.01) A61K 31/4725 (2006.01) A61K 31/497 (2006.01) A61K 31/519 (2006.01) A61P 3/00 (2006.01)
Patent
CA 2691425
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.
La présente invention concerne l'utilisation de types spécifiques de composés agissant en tant que chaperons pharmacologiques qui présentent une activité stabilisatrice de l'enzyme phénylalanine hydroxylase (PAH) et sont destinés à la prophylaxie et/ou au traitement de l'hyperphénylalaninémie (HPA) et de la phénylcétonurie (PCU) ainsi que de maladies, de troubles et de pathologies connexes. La présente invention concerne en outre des compositions pharmaceutiques contenant les composés selon la présente invention ainsi que des procédés de traitement de la HPA, en particulier, de la PCU ainsi que de maladies, de troubles et de pathologies connexes.
Cremades Casasin Nunilo
Martinez Ruiz Aurora
Pey Rodriguez Angel
Sancho Sanz Javier
Velazquez Campoy Adrian
Gowling Lafleur Henderson Llp
Universidad de Zaragoza
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