Compositions for the treatment of hyperphenylalaninemia
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United States of America Patent
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Dec 25, 2012
Grant Date -
Nov 11, 2010
app pub date -
Jun 27, 2008
filing date -
Jun 28, 2007
priority date (Note) -
Expired
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Abstract
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.
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