PTC Therapeutics will need to carry out at least one further trial to demonstrate the effectiveness of its Duchenne Muscular Dystrophy therapy Translarna if it is to have a chance of bagging its approval in the US.
Muscular Dystrophy UK is campaigning for faster approval and funding for therapies for children and adults with muscle-wasting treatments, and warns that the wait for a government response to the Accelerated Access Review “adds to delays to new drugs”.