A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 48/00 (2006.01) A61K 31/7036 (2006.01) A61K 31/785 (2006.01) A61K 38/10 (2006.01) A61K 38/47 (2006.01) A61K 38/48 (2006.01) A61P 3/00 (2006.01)
Patent
CA 2438750
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.
L'invention concerne une méthode pour traiter des maladies lysosomales telles que le syndrome de Hurler et la maladie de Batten Steinert chez des individus ayant besoin d'un tel traitement. Cette méthode comprend l'étape consistant à administrer à ces individus une dose efficace au niveau thérapeutique d'un aminoglycoside. Par ailleurs, cette méthode peut également comprendre le traitement de l'individu à l'aide d'une enzymothérapie de remplacement. En outre, cette invention concerne une méthode permettant de supprimer pharmaceutiquement des mutations stop prématurées chez un individu avec ces maladies lysosomales, laquelle méthode comprend l'étape consistant à administrer audit individu une dose efficace au niveau pharmacologique d'un aminoglycoside.
Bedwell David M.
Keeling Kim M.
Borden Ladner Gervais Llp
Uab Research Foundation
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