Claims for Patent: 12,042,488
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Summary for Patent: 12,042,488
| Title: | Methods of treating Fabry patients having renal impairment |
| Abstract: | Provided are methods for treatment of Fabry disease in patients having HEK assay amenable mutations in α-galactosidase A. Certain methods comprise administering migalastat or a salt thereof every other day, such as administering about 150 mg of migalastat hydrochloride every other day. |
| Inventor(s): | Jeff Castelli, Elfrida Benjamin |
| Assignee: | Amicus Therapeutics Inc |
| Application Number: | US18/326,274 |
| Patent Claims: |
1. An α-galactosidase A protein having a HEK amenable mutation selected from the group consisting of: G334E, N34D and p.V254del, wherein the protein is bound to migalastat. 2. The α-galactosidase A protein of claim 1, wherein the mutation is G334E. 3. The α-galactosidase A protein of claim 1, wherein the mutation is N34D. 4. The α-galactosidase A protein of claim 1, wherein the mutation is p.V254del. 5. The α-galactosidase A protein of claim 1, wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation. 6. The α-galactosidase A protein of claim 5, wherein the mutation is G334E. 7. The α-galactosidase A protein of claim 5, wherein the mutation is N34D. 8. The α-galactosidase A protein of claim 5, wherein the mutation is p.V254del. 9. An α-galactosidase A protein having a HEK amenable mutation N215I, wherein the protein is bound to migalastat. 10. The α-galactosidase A protein of claim 9, wherein the protein bound to migalastat has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation. |
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