Aminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathy
Editorial Group: Cochrane Neuromuscular Disease Group
Published Online: 8 JUL 2009
Copyright © 2009 The Cochrane Collaboration. Published by John Wiley & Sons, Ltd.
How to Cite
Sampson JB, Vardeny O, Flanigan KM. Aminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathy (Protocol). Cochrane Database of Systematic Reviews 2009, Issue 3. Art. No.: CD007985. DOI: 10.1002/14651858.CD007985.
- Publication Status: New
- Published Online: 8 JUL 2009
This is the protocol for a review and there is no abstract. The objectives are as follows:
To determine whether aminoglycoside treatment or related, nonsense mutation-supressing drug treatment of dystrophinopathy patients with premature stop codon mutations in the DMD gene (1) results in stabilization or improvement in function, (2) results in increased expression of dystrophin protein in muscle, or (3) is sufficiently safe to be considered as a treatment option.