Patents
US 12414944 B2: Methods of treating fabry disease in patients having the G9331A mutation in the GLA gene
- Granted
- 2025-09-16
- Filed
- 2022-01-31 application 17588943
- Type
- Utility · 12 claims
- Elsewhere
- Google Patents ↗ · USPTO patent document ↗ · USPTO Patent Center record ↗
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.
Source: PVGPATDIS, file of 2026-04-10 (ipg250916.xml).
Assignees
| Assignee | Type | Place | Also on furp.org | Source |
|---|---|---|---|---|
| Amicus Therapeutics, Inc. 158 patents | US company or organization | Philadelphia, PA | AMICUS THERAPEUTICS, INC. Form D issuer EXACT high confidence: identical full name and state AMICUS THERAPEUTICS, INC. SEC filer EXACT medium confidence: identical full name, one of each nationally | PVGPATDIS, file of 2026-04-10 |
Inventors
| Inventor | Place | Patents | Person page | Source |
|---|---|---|---|---|
| Elfrida Benjamin | Millstone Township, NJ | 57 | PVGPATDIS, file of 2026-04-10 |
CPC categories
| Subclass | Category | First group | Source |
|---|---|---|---|
| A61K | Preparations for medical, dental or toiletry purposes | A61K31/445 | PVGPATDIS, file of 2026-04-10 |
| A61P | Specific therapeutic activity of chemical compounds or medicinal preparations | A61P43/00 | PVGPATDIS, file of 2026-04-10 |
Cooperative Patent Classification, as currently assigned (one row per subclass).
Assignments and security interests
| Recorded | What | From | To | Reel/frame | Source |
|---|---|---|---|---|---|
| 2026-04-27 | Release of security interest Release of security interest | WILMINGTON TRUST, NATIONAL ASSOCIATION signed 2026-04-27 | AMICUS THERAPEUTICS, INC. PHILADELPHIA, PA | 75494/30 | PASDL, file of 2026-08-13 |
| 2026-04-27 | Security interest Security interest Security interest | BIOMARIN PHARMACEUTICAL INC. signed 2026-04-27 AMICUS THERAPEUTICS, INC. signed 2026-04-27 | CITIBANK, N.A., AS COLLATERAL AGENT NEW YORK, NY | 75493/968 | PASDL, file of 2026-10-15 |
| 2023-10-06 | Security interest Security interest Security interest | AMICUS THERAPEUTICS, INC. signed 2023-10-05 | WILMINGTON TRUST, NATIONAL ASSOCIATION MINNEAPOLIS, MN | 65177/196 | PASDL, file of 2026-10-15 |
Documents recorded with the USPTO Assignment Recordation Branch that list this patent. A security interest is a lender’s claim on the patent as collateral; a release ends it.
USPTO bulk data: PatentsView granted patents, disambiguated (PVGPATDIS) for patents, assignees, inventors, places and CPC categories; the USPTO Patent Assignment Dataset (ECORSEXC) for assignments and security interests. Patents filed in the last 20 years. No claims or full text. Links to other records are by name (and state): EXACT means the identical full name and state; FUZZY means a looser name match, with the confidence shown.