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Aminoglycosides and other nonsense suppression therapies for the treatment of dystrophinopathy

  • Protocol
  • Intervention

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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.