Low-Dose Ataluren Shows Some Benefit in DMD/BMD

A new analysis of a trial of ataluren in DMD/BMD due to nonsense mutations shows a low dose may slow decline in walking ability

• A phase 2b, 174-person trial of ataluren in DMD/BMD due to nonsense mutations showed the low-dose regimen, but not the high-dose regimen, resulted in a longer distance walked at the end of 48 weeks than the placebo.
  
• Those on the lower dose walked an average of 29.7 meters (about 97 feet) more in six minutes than the high-dose or placebo groups, although all groups' walking distance declined over the course of the trial.
  
• The findings make it more likely than did an initial analysis that development of ataluren for nonsense-mutation-related DMD/BMD will continue.
  
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