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):	Kaye; Edward M. (Cambridge, MA)
Assignee:	Sarepta Therapeutics, Inc. (Cambridge, MA)
Application Number:	14/214,567
Patent Claims:	1. An oral methylphenidate HCl solution comprising: about 0.1 mg/ml to about 10.0 mg/ml methylphenidate HCl; and about 0.5 mg/ml to about 5.0 mg/ml of at least one organic acid, the methylphenidate HCl and the at least one organic acid being dissolved in a solvent system, the solvent system comprising: about 10% to about 45% water; about 30% to about 70% of at least one polyol solvent; about 10% to about 70% of at least one glycol solvent; and wherein the oral methylphenidate HCl solution is storage stable. 2. The methylphenidate HCl solution according to claim 1, wherein the at least one organic acid is selected from the group consisting of acetic acid, ascorbic acid, citric acid, fumaric acid, malic acid, succinic acid, tartaric acid and mixtures thereof. 3. The methylphenidate HCL solution according to claim 1, wherein the at least one polyol solvent is selected from the group consisting of glycerin, sorbitol, sucrose, fructose and mixtures thereof. 4. The methylphenidate HCl solution according to claim 1, wherein the at least one glycol solvent is selected from the group consisting of propylene glycol, polyalkylene glycol products and mixtures thereof. 5. The methylphenidate HCl solution according to claim 1, further including at least one pharmaceutical additive selected from the group consisting of flavorings, colorants, buffers, preservatives and mixtures thereof. 6. An oral methylphenidate HCl solution comprising: about 0.1 mg/ml to about 10.0 mg/ml methylphenidate HCl; and about 0.5 mg/ml to about 3.0 mg/ml of at least one organic acid, the methylphenidate HCl and the at least one organic acid being dissolved in a solvent system, the solvent system comprising: about 10% to about 45% water; about 40% to about 60% of at least one polyol solvent; about 10% to about 30% of at least one glycol solvent; and wherein the oral methylphenidate HCl solution is storage stable. 7. The methylphenidate HCl solution according to claim 6, wherein at least one organic acid is selected from the group consisting of acetic acid, ascorbic acid, citric acid, fumaric acid, malic acid, succine acid, tartaric acid and mixtures thereof. 8. The methylphenidate HCl solution according to claim 6, wherein the at least one polyol solvent is selected from the group consisting of glycerin, sorbitol, sucrose, fructose and mixtures thereof. 9. The methylphenidate HCl solution according to claim 6, wherein the at least one glycol solvent is selected from the group consisting of propylene glycol, polyalkylene glycol products and mixtures thereof. 10. The methylphenidate HCl solution according to claim 6, further including at least one pharmaceutical additive selected from the group consisting of flavorings, colorants, buffers, preservatives and mixtures thereof. 11. An oral methylphenidate HCl solution comprising: about 0.1 mg/ml to about 10.0 mg/ml methylphenidate HCl; and about 0.5 mg/ml to about 1.5 mg/ml of at least one organic acid, the methylphenidate HCl and the at least one organic acid being dissolved in a solvent system, the solvent system comprising: about 30% to about 40% water; about 45% to about 55% of at least one polyol solvent; about 10% to about 20% of at least one glycol solvent; and wherein the oral methylphenidate HCl solution is storage stable. 12. The methylphenidate HCl solution according to claim 11, wherein the at least one organic acid includes citric acid. 13. The methylphenidate HCl solution according to claim 11, wherein the at least one polyol solvent includes glycerin. 14. The methylphenidate HCl solution according to claim 11, wherein the at least one glycol solvent includes polyethylene glycol. 15. The methylphenidate HCl solution according to claim 11, further including at least one pharmaceutical additive selected from the group consisting of flavorings, colorants, buffer, preservatives and mixtures thereof.