AMINOGLYCOSIDE TREATMENT FOR LYSOSOMAL STORAGE DISEASES

Title:

AMINOGLYCOSIDE TREATMENT FOR LYSOSOMAL STORAGE DISEASES

Document Type and Number:

WIPO Patent Application WO2002065989

Kind Code:

A3

Abstract:

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 method 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.

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Inventors:

BEDWELL DAVID M (US)
KEELING KIM M (US)

Application Number:

PCT/US2002/004842

Publication Date:

December 12, 2002

Filing Date:

February 20, 2002

Export Citation:

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Assignee:

UAB RESEARCH FOUNDATION (US)
BEDWELL DAVID M (US)
KEELING KIM M (US)

International Classes:

A61K31/7008; C07H15/224; A61K31/7036; A61K31/704; A61K35/76; A61K38/00; A61K38/46; A61K38/47; A61K38/48; A61K48/00; A61P3/00; A61P31/00; A61P43/00; C07H15/236; (IPC1-7): A61K31/70

Foreign References:

US5310646A

1994-05-10

Other References: