C - Chemistry – Metallurgy – 12 – Q
Patent
C - Chemistry, Metallurgy
12
Q
C12Q 1/02 (2006.01) A61K 31/445 (2006.01) A61K 31/45 (2006.01) A61K 31/452 (2006.01) C12Q 1/34 (2006.01) G01N 33/50 (2006.01) G01N 33/68 (2006.01)
Patent
CA 2715407
The present invention provides methods to determine whether a patient with a lysosomal storage disorder will benefit from treatment with a specific pharmacological chaperone. The present invention exemplifies an in vitro method for determining .alpha.-galactosidase A responsiveness to a pharmacological chaperone such as 1-deoxygalactonojirimycin in a cell line expressing a mutant from of .alpha.- galactosidase A. The invention also provides a method for diagnosing Fabry disease in patients suspected of having Fabry disease.
Cette invention concerne des méthodes permettant de déterminer si un patient atteint dun déficit du stockage lysosomal tirera les bénéfices dun traitement avec un chaperon moléculaire pharmacologique spécifique. Linvention donne en exemple une méthode in vitro permettant de déterminer la réponse de la-galactosidase A à un chaperon moléculaire pharmacologique comme la 1-désoxygalactonojirimycine dans une lignée cellulaire exprimant un mutant de la- galactosidase A. Linvention concerne également une méthode permettant de diagnostiquer la maladie de Fabry chez des patients suspectés den souffrir.
Benjamin Elfrida
Do Hung V.
Flanagan John
Wu Xiaoyang
Wustman Brandon
Amicus Therapeutics Inc.
Ridout & Maybee Llp
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