Systemic gene replacement therapy for treatment of X-linked myotubular myopathy (XLMTM)
US8957044B2 · kind B2 · utility
6Cited by
10References
15Claims
0Family size
Assignees
Inventors
Key dates
| Filing date | Feb 28, 2014 |
| Grant date | Feb 17, 2015 |
| Priority date | — |
| Expiry date | Feb 28, 2034 |
Classification
- Technology area (CPC C)Chemistry; Metallurgy
- CPC primaryC12N2750/14143
- WIPO fieldPharmaceuticals
- WIPO sectorChemistry
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.
Source: USPTO / EPO open patent data. Objective bibliographic and citation counts.