CA2715407C — Method to predict response to pharmacological chaperone treatment of diseases
Assigned to Amicus Therapeutics Inc · Expires 2022-07-26 · 4y expired
What this patent protects
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 responsive…
USPTO Abstract
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.
Drugs covered by this patent
Bibliographic data sourced from FDA Orange Book + USPTO public records. Plain-English summary generated by AI grounded in source text. Patent term extensions (PTR, SPC, pediatric) may shift the effective expiry. Not legal advice.
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