The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Dec. 31, 2024
Filed:
Jul. 22, 2020
Wake Forest University Health Sciences, Winston-Salem, NC (US);
Genethon, Evry, FR;
Children's Medical Center Corporation, Boston, MA (US);
Martin K. Childers, Seattle, WA (US);
Alan H. Beggs, Needham, MA (US);
Ana Maria Buj Bello, Paris, FR;
Wake Forest University Health Sciences Genethon, Evry, FR;
Children's Medical Center Corporation, Boston, MA (US);
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.