Use of non-glucocorticoid steroids for the treatment of...

A - Human Necessities – 61 – K

Patent

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A61K 31/56 (2006.01) A61P 21/00 (2006.01)

Patent

CA 2574135

The invention relates to the therapeutic use of certain classes of steroid compounds for treatment of muscular diseases, in particular muscle diseases caused by mutations in the gene encoding for dystrophin (Duchenne Muscular Dystrophy, DMD, and Becker Muscular Dystrophy, BMD). The steroid compounds increase the levels of the dystrophin-related protein utrophin in cultured human muscle cells derived from donors affected with Duchenne Muscular Dystrophy.

Cette invention concerne l'utilisation à des fins thérapeutiques de certaines classes de composés stéroïdes pour le traitement de maladies musculaires, en particulier de maladies musculaires provoquées par des mutations intervenant dans le gène codant pour la dystrophine (dystrophie musculaire de Duchenne (DMD), et la dystrophie musculaire de Becker (BMD)). Ces composés stéroïdes augmentent les niveaux de l'utrophine, une protéine associée à la dystrophine, dans des cellules musculaires humaines cultivées provenant de donneurs affectés par la dystrophie musculaire de Duchenne.

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