Intervention Protocol

Aminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathy

  1. Jacinda B Sampson1,*,
  2. Orly Vardeny2,
  3. Kevin M Flanigan3

Editorial Group: Cochrane Neuromuscular Disease Group

Published Online: 8 JUL 2009

DOI: 10.1002/14651858.CD007985


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.

Author Information

  1. 1

    University of Utah, Department of Neurology, Salt Lake City, Utah, USA

  2. 2

    University of Wisconsin, School of Pharmacy, Madison, Wisconsin, USA

  3. 3

    University of Utah, Department of Neurology and Department of Human Genetics, Salt Lake City, Utah, USA

*Jacinda B Sampson, Department of Neurology, University of Utah, Clinical Neurosciences Center, 175 North Medical Drive East, 5th Floor, Salt Lake City, Utah, 84132, USA. jacinda.sampson@hsc.utah.edu.

Publication History

  1. Publication Status: New
  2. Published Online: 8 JUL 2009

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Abstract

  1. Top of page
  2. Abstract

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.