Systemic gene replacement therapy for treatment of X-linked MyoTubular Myopathy (XLMTM)
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United States of America Patent
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Aug 16, 2016
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Jan 13, 2015
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Mar 1, 2013
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Abstract
The present invention provides compositions and methods for treating a myopathy. In certain embodiments, the invention provides compositions and methods for treating, improving muscle function, and prolonging survival in a subject with X-linked myotubular myopathy (XLMTM). The present invention provides a method comprising systemic administration of a composition that induces the increased expression of myotubularin in the muscle of a subject. The invention provides sustained regional and global increases in muscle function.
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