http://rdf.ncbi.nlm.nih.gov/pubchem/patent/EP-2142197-A2

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assignee http://rdf.ncbi.nlm.nih.gov/pubchem/patentassignee/MD5_3386f5906dc1bdc9c934203594712c66
classificationCPCInventive http://rdf.ncbi.nlm.nih.gov/pubchem/patentcpc/A61K38-47
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classificationIPCInventive http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61P43-00
http://rdf.ncbi.nlm.nih.gov/pubchem/patentipc/A61K33-24
filingDate 2008-03-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_bf44362328376a83bd312934669801fd
publicationDate 2010-01-13-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber EP-2142197-A2
titleOfInvention Method for the treatment of fabry disease using pharmacological chaperones
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.
priorityDate 2007-03-30-04:00^^<http://www.w3.org/2001/XMLSchema#date>
type http://data.epo.org/linked-data/def/patent/Publication

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