Methods for treating muscular dystrophy with casimersen
AssigneeSarepta Therapeutics, Inc.
InventorEdward M. KAYE
Life of this patent
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Abstract
The present disclosure provides, among other things, improved compositions and methods for treating muscular dystrophy. For example, the disclosure provides methods for treating Duchenne muscular dystrophy patients having a mutation in the DMD gene that is amenable to exon 45 skipping by administering an effective amount of casimersen.
Claims22
1-18. (canceled)
A method for increasing dystrophin production in a human pediatric patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 45 skipping, comprising administering by injection to the human pediatric patient a dose of casimersen, or a pharmaceutically acceptable salt thereof once a week, for at least 48 weeks, wherein the administering results in:
- an increase in skipping exon 45 compared to baseline exon 45 skipping prior to beginning said administering, and
- a statistically significant increase in dystrophin production compared to baseline dystrophin production prior to beginning said administering.
20-26. (canceled)
A method for treating Duchenne muscular dystrophy (DMD) in a human pediatric patient in need thereof who has a mutation of the DMD gene that is amenable to exon 45 skipping, comprising administering by injection to the human pediatric patient a dose of casimersen, or a pharmaceutically acceptable salt thereof once a week, for at least 48 weeks, wherein the administering results in:
- an increase in skipping exon 45 compared to baseline exon 45 skipping prior to beginning said administering, and
- a statistically significant increase in dystrophin production compared to baseline dystrophin production prior to beginning said administering.
A method for restoring an mRNA reading frame to induce exon skipping in a human pediatric patient with Duchenne muscular dystrophy (DMD) in need thereof who has a mutation of the DMD gene that is amenable to exon 45 skipping, comprising administering by injection to the human pediatric patient a dose of casimersen, or a pharmaceutically acceptable salt thereof once a week, for at least 48 weeks, wherein the administering results in:
- an increase in skipping exon 45 compared to baseline exon 45 skipping prior to beginning said administering, and
- a statistically significant increase in dystrophin production compared to baseline dystrophin production prior to beginning said administering.
Dependencies are inferred from the claim text.