Methods of treating Fabry patients having renal impairment

1. A method of treating Fabry disease, the method comprising administering migalastat to a patient in need thereof, wherein the patient has an a -galactosidase A protein comprising a HEK assay amenable mutation selected from the group consisting of A13T, A15T, W24R, N34T, M42K, L54F, P60T, P60S, E87D, L89F, R112G, Y123C, H125L, I133M, K140T, F145S, P146R, Y152H, D165G, p.M187_S188dup, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, S304N, D322N, G325R, K326N, G334E, E358Q, E358D, G361E, G375E, T412N and M421V.

2. The method of claim 1 , wherein the mutation is selected from the group consisting of: A13T, A15T, W24R, N34T, M42K, L54F, P60T, P60S, E87D, L89F, I133M, K140T, F145S, P146R, Y152H, D165G, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, S304N, D322N, G325R, K326N, G334E, E358Q, E358D, G361E, G375E, T412N and M421V.

3. The method of claim 1 , wherein the mutation is selected from the group consisting of: L54F, P60T, P60S, I133M, K140T, F145S, V199G, I219L, Q221P, Q250R, G261C, G271D, W277G, M284V, R301L and I303F.

4. The method of claim 1 , wherein the mutation is selected from the group consisting of: A15T, N34T, M42K, L54F, P60T, P60S, L89F, R112G, Y123C, H125L, I133M, K140T, F145S, P146R, D165G, p.M187_S188dup, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, S304N, G325R, E358Q, E358D, G361E, G375E, T412N and M421V.

5. The method of claim 1 , wherein the mutation is selected from the group consisting of: N34T, M42K, L54F, P60T, P60S, L89F, R112G, Y123C, H125L, I133M, K140T, F1455, P146R, D165G, p.M187_S188dup, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, G325R, E358Q, E358D, G361E, G375E and T412N.

6. The method of claim 1 , wherein the mutation is selected from the group consisting of: N34T, R112G, G271D, W287L, G325R and T412N.

7. The method of claim 1 , wherein the mutation is selected from the group consisting of: A15T, N34T, M42K, L54F, P60T, P60S, L89F, I133M, K140T, F145S, P146R, D165G, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, S304N, G325R, E358Q, E358D, G361E, G375E, T412N and M421V.

8. The method of claim 1 , wherein the mutation is selected from the group consisting of: N34T, M42K, L54F, P60T, P60S, L89F, I133M, K140T, F145S, P146R, D165G, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, G325R, E358Q, E358D, G361E, G375E and T412N.

9. The method of claim 1 , wherein the mutation is selected from the group consisting of: N34T, E87D, I133M, F145S, P146R, Y152H, D165G, M208R, I219L, Q221P, N224T, Q250R, G261C, I303F, K326N, E358Q and G375E.

10. The method of claim 1 , wherein the mutation is selected from the group consisting of: N34T, I133M, F145S, P146R, D165G, M208R, I219L, Q221P, N224T, Q250R, G261C, I303F, E358Q and G375E.

11. The method of claim 1 , wherein the mutation is selected from the group consisting of: I133M, F145S, I219L, Q221P, Q250R, G261C, and I303F.

12. The method of claim 1 , wherein the mutation is selected from the group consisting of: R112G and G325R.

13. The method of claim 1 , wherein the mutation is selected from the group consisting of: A13T, A15T, W24R, N34T, M42K, L54F, P60T, P60S, E87D, L89F, Y123C, H125L, I133M, K140T, F145S, P146R, Y152H, D165G, p.M187_S188dup, V199G, M208R, I219L, Q221P, N224T, Q250R, G261C, G271D, W277G, W277C, M284V, R301L, I303F, S304N, D322N, K326N, G334E, E358Q, E358D, G361E, G375E, T412N and M421V.

14. The method of claim 1 , wherein the patient has a Fabry disease-causing mutation.

15. The method of claim 1 , wherein the migalastat or salt thereof is administered to the patient every other day.

16. The method of claim 1 , wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof every other day.

17. The method of claim 1 , wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.

18. The method of claim 1 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

19. The method of claim 1 , wherein the patient is male.

20. The method of claim 1 , wherein the patient is female.

21. The method of claim 1 , wherein the patient has renal impairment.

22. The method of claim 1 , wherein the patient has mild or moderate renal impairment.

23. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient.

24. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.

25. The method of claim 1 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.

26. The method of claim 1 , wherein the patient has a proteinuria level of less than 100 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

27. The method of claim 1 , wherein the patient has a proteinuria level of 100 to 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

28. The method of claim 1 , wherein the patient has a proteinuria level of greater than 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

29. The method of claim 1 , wherein the migalastat or salt thereof is administered orally.

30. The method of claim 29 , wherein the migalastat or salt thereof is in a solid dosage form.

31. The method of claim 30 , wherein the solid dosage form comprises a capsule.

32. The method of claim 1 , wherein the migalastat is administered as a pharmaceutically acceptable salt.

33. The method of claim 8 , wherein the patient has a Fabry disease-causing mutation.

34. The method of claim 8 , wherein the migalastat or salt thereof is administered to the patient every other day.

35. The method of claim 8 , wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof every other day.

36. The method of claim 8 , wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.

37. The method of claim 8 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

38. The method of claim 8 , wherein the patient is male.

39. The method of claim 8 , wherein the patient is female.

40. The method of claim 8 , wherein the patient has renal impairment.

41. The method of claim 40 , wherein the patient has mild or moderate renal impairment.

42. The method of claim 8 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient.

43. The method of claim 8 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.

44. The method of claim 8 , wherein the patient is an enzyme replacement therapy (ERT)-naive patient.

45. The method of claim 8 , wherein the patient has a proteinuria level of less than 100 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

46. The method of claim 8 , wherein the patient has a proteinuria level of 100 to 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

47. The method of claim 8 , wherein the patient has a proteinuria level of greater than 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

48. The method of claim 8 , wherein the migalastat or salt thereof is administered orally.

49. The method of claim 48 , wherein the migalastat or salt thereof is in a solid dosage form.

50. The method of claim 49 , wherein the solid dosage form comprises a capsule.

51. The method of claim 8 , wherein the migalastat is administered as a pharmaceutically acceptable salt.

52. The method of claim 9 , wherein the patient has a Fabry disease-causing mutation.

53. The method of claim 9 , wherein the migalastat or salt thereof is administered to the patient every other day.

54. The method of claim 9 , wherein the patient is administered about 123 to about 300 mg of the migalastat or salt thereof every other day.

55. The method of claim 9 , wherein the patient is administered about 150 mg of the migalastat or salt thereof every other day.

56. The method of claim 9 , wherein the patient is administered about 150 mg of migalastat hydrochloride every other day.

57. The method of claim 9 , wherein the patient is male.

58. The method of claim 9 , wherein the patient is female.

59. The method of claim 9 , wherein the patient has renal impairment.

60. The method of claim 39 , wherein the patient has mild or moderate renal impairment.

61. The method of claim 9 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient.

62. The method of claim 9 , wherein the patient is an enzyme replacement therapy (ERT)-experienced patient with renal impairment.

63. The method of claim 9 , wherein the patient is an enzyme replacement therapy (ERT)-naïve patient.

64. The method of claim 9 , wherein the patient has a proteinuria level of less than 100 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

65. The method of claim 9 , wherein the patient has a proteinuria level of 100 to 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

66. The method of claim 9 , wherein the patient has a proteinuria level of greater than 1,000 mg/24 hr prior to initiating the administration of the migalastat or salt thereof.

67. The method of claim 9 , wherein the migalastat or salt thereof is administered orally.

68. The method of claim 67 , wherein the migalastat or salt thereof is in a solid dosage form.

69. The method of claim 68 , wherein the solid dosage form comprises a capsule.

70. The method of claim 9 , wherein the migalastat is administered as a pharmaceutically acceptable salt.