A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 31/452 (2006.01) A61P 3/00 (2006.01) G01N 33/50 (2006.01)
Patent
CA 2682441
The present invention provides a method treating a patient with Fabry disease by determining whether there is an improvement of a surrogate marker that is associated with Fabry disease following administration of a specific pharmacological chaperone of .alpha.-galactosidase A. The method includes administering an effective amount of 1-deoxygalactonojirimycn to the individual, wherein the 1- deoxygalactonojirimycin binds to alpha-galactosidase A in an amount effective to increase activity of the alpha-galactosidase A. The present invention also provides a method for monitoring and increasing a therapeutic response of a patient with Fabry disease following administration of a specific pharmacological chaperone of .alpha.- galactosidase A by evaluating the effect on the cytoplasmic staining pattern of a cell from the patient, wherein detection of a staining pattern in the cell that is similar to the staining pattern in a cell from a healthy individual indicates that the individual with Fabry disease is a responder.
La présente invention propose un procédé de traitement d'un patient atteint de la maladie de Fabry en déterminant s'il y a amélioration d'un marqueur de substitution qui est associé à la maladie de Fabry à la suite de l'administration d'un chaperon pharmacologique spécifique de a-galactosidase A. Le procédé comprend l'administration d'une quantité efficace de 1-déoxygalactonojirimycine au sujet, la 1-déoxygalactonojirimycine se liant à l'a-galactosidase A en une quantité efficace pour augmenter l'activité de l'a-galactosidase A. La présente invention propose également un procédé pour surveiller et augmenter la réponse thérapeutique d'un patient atteint de la maladie de Fabry à la suite de l'administration d'un chaperon pharmacologique spécifique de a-galactosidase A en évaluant l'effet sur le motif de coloration cytoplasmique d'une cellule du patient, la détection d'un motif de coloration dans la cellule qui est similaire au motif de coloration dans une cellule provenant d'un individu sain indiquant que l'individu atteint de la maladie de Fabry est un répondeur.
Amicus Therapeutics Inc.
Ridout & Maybee Llp
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