FDA will review resubmission of PTC’s Translarna for Duchenne caused by nonsense mutation

CureDuchenne is pleased to share that PTC Therapeutics has announced that the FDA has accepted for review the New Drug Application (NDA) for Translarna (ataluren) for the treatment of Duchenne caused by nonsense mutations.  The resubmission includes results from a global placebo-controlled study showing significant functional benefits at 72 weeks of treatment, as well as significant long-term benefits captured in the STRIDE registry.  

Link to Press Release HERE

Letter to the community below: