Patent ID: 8951978
Filing Date: 2015-02-10
Classification: A61K,A61P

Abstract:
1. A method of treating a genetic disease, comprising the steps of: identifying a patient, wherein the patient exhibits a pathology that includes the imposition of a stop codon in the open reading frame of an otherwise functional gene, and wherein the pathology is selected from the group consisting of Duchenne Muscular Dystrophy, cystic fibrosis, Hurler's syndrome, and Becker muscular dystrophies; and formulating a mixture of a therapeutically effective dose of an aminoglycoside enriched in an isolated and purified compound having the following formula, or a pharmaceutically acceptable salt thereof: wherein, R 1 and R 3 are H, and R 2 is CH 3 , and wherein the compound is present in said formulation at a level sufficient to reduce the cellular toxicity of the aminoglycoside; and administering a therapeutically effective dose of said formulation to the patient.