PTC

Compounds designed to alter genetic instructions for dystrophin, increase production of utrophin, or prevent scar tissue formation in muscle are moving through the development pipeline

posted on November 5, 2014 - 4:03pm
Drug development for Duchenne muscular dystrophy (DMD) is progressing on several fronts. Here are some updates as of early November 2014. PTC is moving forward with ataluren

After restructuring its agreement with Genzyme, PTC will continue developing ataluren for nonsense-mutation DMD/BMD and will study the drug in former trial participants

posted on September 15, 2011 - 9:24am
Update (July 25, 2012): This story was updated to reflect the fact that PTC has announced its intention to begin an open-label trial of ataluren in DMD/BMD for former ataluren trial participants in Europe, Israel and Australia.