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 of treating Duchenne muscular dystrophy (DMD) in a human subject who has a mutation of the DMD gene that is amenable to exon 51 skipping, comprising administering to the human subject a composition comprising eteplirsen and a phosphate-buffered saline at a dose of eteplirsen of about 30 mg/kg to about 50 mg/kg for a period of time sufficient to increase the number of dystrophin-positive fibers in a subject to at least 20% of normal.
2. The method of claim 1 , wherein eteplirsen is administered for a period of time sufficient to increase the number of dystrophin positive fibers to about 50% of normal.
3. The method of claim 1 , wherein the dose of eteplirsen is about 30 mg/kg.
4. The method of claim 1 , wherein the composition is administered by systemic administration.
5. The method of claim 1 , wherein the composition is administered once weekly by infusion.
6. The method of claim 1 , further comprising administering a steroid to the human subject.
7. The method of claim 6 , wherein the human subject is administered a stable dose of the steroid for at least 24 weeks prior to the first dose of eteplirsen.
8. The method of claim 7 , wherein the steroid is a glucocorticoid.
9. The method of claim 1 , wherein the human subject is 7 to 13 years of age.
10. The method of claim 9 , wherein the human subject is a boy.
11. The method of claim 7 , wherein the human subject is 7 to 13 years of age.
12. The method of claim 11 , wherein the human subject is a boy.