Title:
薬理シャペロンを用いるファブリー病の治療方法
Document Type and Number:
Japanese Patent JP2010523578
Kind Code:
A
Abstract:
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 α-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 α-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.
Inventors:
David Polling
Application Number:
JP2010502218A
Publication Date:
July 15, 2010
Filing Date:
March 28, 2008
Export Citation:
Assignee:
Amicus Therapeutics, Inc.
International Classes:
A61K45/00; A61K31/445; A61P3/00; A61P9/00; A61P9/10; A61P13/12; A61P17/00; C12Q1/34
Attorney, Agent or Firm:
Mitsuo Tanaka
Hiroshi Yamazaki
Masaki Yano
Tsuyoshi Sato
Hiroshi Yamazaki
Masaki Yano
Tsuyoshi Sato