Results from PTC’s ataluren study in DMD published in The Lancet … – Seeking Alpha
The Lancet has just published results from PTC Therapeutics’ (PTCT) ACT DMD study assessing Translarna (ataluren). The primary endpoint was the change from baseline in the six-minute walk test (6MWT), which it failed to beat placebo on. The company says a treatment effect was more likely to seen in patients in the transition phase of disease (baseline 6MWT distance of 300-400 meters).
An Ad Com review in the U.S. is scheduled for October 24.