Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
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United States of America
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Aug 11, 2020
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Dec 27, 2018
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Dec 28, 2017
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Dec 28, 2017
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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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