US11234972 — Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene
Method of Use · Assigned to Amicus Therapeutics Inc · Expires 2037-03-15 · 11y remaining
What this patent protects
This patent protects methods of treating Fabry disease in patients with a specific genetic mutation using a pharmacological chaperone.
USPTO 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 splice site mutation in intron 4 of 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.
Drugs covered by this patent
FDA Patent Use Codes
When a patent is method-of-use, FDA lists it once per applicable indication ("U-code"). Each U-code carves out a specific therapeutic use that generic filers must either license or design around.
| Code | Description | Drug |
|---|---|---|
U-2371 |
— | migalastat-hydrochloride |
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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