Claims for Patent: 9,506,058

Summary for Patent: 9,506,058

Title:	Compositions for treating muscular dystrophy
Abstract:	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.
Inventor(s):	Edward M. Kaye
Assignee:	Sarepta Therapeutics Inc
Application Number:	US14/214,567
Patent Litigation and PTAB cases:	See patent lawsuits and PTAB cases for patent 9,506,058
Patent Claims:	1. A method for treating 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 eteplirsen at a dose of about 30 mg/kg weekly 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 according to claim 1, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen. 4. A method for reducing the loss of ambulation 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 eteplirsen at a dose of about 30 mg/kg weekly for more than 120 weeks, thereby reducing the loss of ambulation relative to baseline, in the patient. 5. The method according to claim 4, wherein the patient is a pediatric patient. 6. The method according to claim 4, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen. 7. A method for restoring an mRNA reading frame to induce dystrophin protein 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 eteplirsen at a dose of about 30 mg/kg weekly for more than 120 weeks, thereby restoring the mRNA reading frame to induce dystrophin protein production in the patient. 8. The method according to claim 7, wherein the patient is a pediatric patient. 9. The method according to claim 7, wherein the patient is administered an oral corticosteroid for at least 24 weeks prior to the first dose of eteplirsen. 10. The method of claim 4, wherein the dystrophin protein production is measured by reverse transcription polymerase chain reaction (RT-PCR), Western blot analysis, or immunohistochemical detection. 11. The method of claim 4, wherein ambulation is measured using the North Star Ambulatory Assessment. 12. A method for treating 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 eteplirsen at a dose of about 30 mg/kg weekly for more than 120 weeks, such that disease progression in the patient is delayed as measured by the 6 Minute Walk Test (6MWT), thereby treating the patient. 13. A method for reducing the loss of ambulation 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 eteplirsen at a dose of about 30 mg/kg weekly for more than 120 weeks, thereby reducing the loss of ambulation relative to baseline in the patient as measured by the 6 Minute Walk Test (6MWT). 14. The method of claim 13, wherein the patient loses less than 5% ambulation, relative to baseline, as measured by the 6 Minute Walk Test (6MWT) by 120 weeks. 15. The method of claim 13, wherein the patient loses no greater than 13.9 meters walking distance relative to baseline, as measured by the 6 Minute Walk Test (6MWT) by 120 weeks. 16. The method of claim 13, wherein the patient maintains ambulation for more than 1.5 years following treatment. 17. A method for reducing loss of pulmonary function 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 eteplirsen at a dose of 30 mg/kg weekly for more than 120 weeks, thereby reducing the loss of pulmonary function in the patient relative to baseline. 18. The method of claim 17, wherein pulmonary function is measured as Maximum Inspiratory Pressure (MIP). 19. The method of claim 17, wherein respiratory muscle function in the patient improves at least 14.6% relative to baseline pulmonary function as measured as Maximum Inspiratory Pressure (MIP). 20. The method of claim 17, wherein pulmonary function is measured as Maximum Expiratory Pressure (MEP). 21. The method of claim 17, wherein respiratory muscle function in the patient improves at least 15% relative to baseline pulmonary function as measured as Maximum Expiratory Pressure (MEP). 22. The method of claim 17, wherein pulmonary function is measured as Forced Vital Capacity (FVC). 23. The method of claim 1, wherein the patient is 7 years of age or older. 24. The method of claim 1, further comprising administering to the patient a corticosteroid. 25. The method of claim 24, wherein the corticosteroid is Betamethasone, Budesonide, Cortisone, Dexamethasone, Hydrocortisone, Methylprednisolone, Prednisolone, or Prednisone. 26. The method of claim 24, wherein the corticosteroid is administered prior to, in conjunction with, or subsequent to administration of eteplirsen. 27. The method of claim 1, further comprising confirming that the patient has a mutation in the DMD gene that is amenable to exon 51 skipping. 28. A method for treating 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 eteplirsen at a dose of about 30 mg/kg weekly for more than 120 weeks to thereby treat DMD, wherein by 120 weeks of treatment respiratory muscle function in the patient improves at least 14.6% relative to baseline pulmonary function as measured as Maximum Inspiratory Pressure (MIP). 29. A method for treating 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 eteplirsen at a dose of about 30 mg/kg weekly for more than 120 weeks to thereby treat DMD, wherein by 120 weeks of treatment respiratory muscle function in the patient improves at least 15% relative to baseline pulmonary function as measured as Maximum Expiratory Pressure (MEP).