Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)

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United States of America Patent

PATENT NO 9895426
SERIAL NO

15384976

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Abstract

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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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Patent Owner(s)

  • CHILDREN'S MEDICAL CENTER CORPORATION;WAKE FOREST UNIVERSITY HEALTH SCIENCES;GENETHON

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Beggs, Alan H Needham, US 8 25
Buj, Bello Ana Maria Paris, FR 13 44
Childers, Martin K Seattle, US 9 38

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