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filingDate 2021-03-05-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_b98244336606864292e231ffc9c44f23
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publicationDate 2021-09-10-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2021178737-A1
titleOfInvention Methods of treating fabry disease in patients having a mutation in the gla gene
abstract Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease. Certain methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for α-galactosidase A, wherein the patient has a mutation in the nucleic acid sequence encoding α-galactosidase A. Also described are uses of pharmacological chaperones for the treatment of Fabry disease and compositions for use in the treatment of Fabry disease.
priorityDate 2020-03-06-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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