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Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
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机译:全身性基因替代疗法,用于治疗X连锁型肌管肌病(XLMTM)
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摘要
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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