AMINOGLYCOSIDE TREATMENT FOR LYSOSOMAL STORAGE DISEASES

摘要

The present invention provides a method of treating lysomal storage diseases such as Hurler syndrome and Batten disease in individuals in need of such treatment, comprising the step of administering to said individuals a therapeutically effective dose of an aminoglycoside. In addition, this metho d may further comprise treating the individual with enzyme replacement therapy . Furthermore, the present invention rovides method of pharmaceutically suppressing premature stop mutations in an indidual with these lysosomal storage diseases, comprising the step of administering to said individual a pharmacologically effective dose of an aminoglycoside.