Methods of treating Fabry patients having renal impairment
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.
1. A molecule comprising migalastat bound to an α-galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of A13P, L89F, Y200C, G271D, W277C and F337S.
2. The molecule of claim 1 , wherein the mutation is A13P.
3. The molecule of claim 1 , wherein the mutation is L89F.
4. The molecule of claim 1 , wherein the mutation is Y200C.
5. The molecule of claim 1 , wherein the mutation is G271D.
6. The molecule of claim 1 , wherein the mutation is W277C.
7. The molecule of claim 1 , wherein the mutation is F337S.
8. The molecule of claim 1 , wherein the mutation is a disease-causing mutation.
9. An α-galactosidase A protein having a HEK amenable mutation selected from the group consisting of A13P, L89F, Y200C, G271D, W277C and F337S
wherein the protein has increased stability as compared to a naturally-occurring α-galactosidase A protein having the same mutation.
10. The α-galactosidase A protein of claim 9 , wherein the protein is bound to migalastat.
11. The α-galactosidase A protein of claim 9 , wherein the mutation is A13P.
12. The α-galactosidase A protein of claim 9 , wherein the mutation is L89F.
13. The α-galactosidase A protein of claim 9 , wherein the mutation is Y200C.
14. The α-galactosidase A protein of claim 9 , wherein the mutation is G271D.
15. The α-galactosidase A protein of claim 9 , wherein the mutation is W277C.
16. The α-galactosidase A protein of claim 9 , wherein the mutation is F337S.
17. The α-galactosidase A protein of claim 9 , wherein the mutation is a disease-causing mutation.