Compositions for treating muscular dystrophy
View Patent ↗Improved compositions and methods for treating muscular dystrophy by administering antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping are described.
1. A method for treating Duchenne muscular dystrophy (DMD) in a patient in need thereof who has a mutation of the DMD gene that is amenable to exon 51 skipping, comprising intravenously administering to the patient a composition comprising eteplirsen, or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier, wherein eteplirsen, or a pharmaceutically acceptable salt thereof, is administered at a dose of about 30 mg/kg once a week for more than 120 weeks, such that disease progression in the patient is delayed, thereby treating the patient.
2. The method according to claim 1 , wherein the patient is a pediatric patient.
3. The method of claim 2 , wherein the patient is 7 years of age or older.
4. The method according to claim 1 , wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen, or a pharmaceutically acceptable salt thereof.
5. The method according to claim 4 , wherein the patient is a pediatric patient.
6. The method of claim 1 , further comprising administering to the patient a corticosteroid.
7. The method of claim 6 , wherein the corticosteroid is Betamethasone, Budesonide, Cortisone, Dexamethasone, Hydrocortisone, Methylprednisolone, Prednisolone, or Prednisone.
8. The method according to claim 6 , wherein the patient is a pediatric patient.
9. The method of claim 6 , wherein the corticosteroid is administered prior to, in conjunction with, or subsequent to administration of eteplirsen, or a pharmaceutically acceptable salt thereof.
10. The method of claim 1 , wherein the composition further comprises a phosphate-buffered saline.
11. A method for restoring an mRNA reading frame to induce dystrophin production in a patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 51 skipping, comprising intravenously administering to the patient a composition comprising eteplirsen, or a pharmaceutically acceptable salt thereof, and a pharmaceutically acceptable carrier, wherein eteplirsen, or a pharmaceutically acceptable salt thereof, is administered at a dose of about 30 mg/kg once a week for more than 120 weeks, such that disease progression in the patient is delayed.
12. The method according to claim 11 , wherein the patient is a pediatric patient.
13. The method of claim 12 , wherein the patient is 7 years of age or older.
14. The method according to claim 11 , wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen, or a pharmaceutically acceptable salt thereof.
15. The method according to claim 14 , wherein the patient is a pediatric patient.
16. The method of claim 11 , further comprising administering to the patient a corticosteroid.
17. The method of claim 16 , wherein the corticosteroid is Betamethasone, Budesonide, Cortisone, Dexamethasone, Hydrocortisone, Methylprednisolone, Prednisolone, or Prednisone.
18. The method according to claim 16 , wherein the patient is a pediatric patient.
19. The method of claim 18 , wherein the patient is 7 years of age or older.
20. The method of claim 17 , wherein the corticosteroid is administered prior to, in conjunction with, or subsequent to administration of eteplirsen, or a pharmaceutically acceptable salt thereof.
21. The method of claim 11 , wherein the pharmaceutically acceptable carrier is a pH buffered solution.
22. The method of claim 21 , wherein the pH buffered solution is a phosphate-buffered saline.