Author Archives: Janet Stewart, MSc

FDA Rejects New Drug Application for Translarna to Treat DMD

The U.S. Food and Drug Administration decided not to approve Translarna (ataluren), by PTC Therapeutics, as a treatment for specific types of Duchenne muscular dystrophy caused by “nonsense” mutations. Although not entirely unexpected, the decision was disappointing to many in the Duchenne community. On Sept. 28, an FDA advisory…