Family with Muscular Dystrophy Heartbroken After Awaiting FDA's Approval on Drug

Controversial aspects of Ataluren

But, not everyone was happy with Ataluren and there are good reasons not to think this is a miraculous drug. Although most children on trial of this medication have shown great results, tests were only focused on their physical performance, being able to walk up and down the stairs and walking a certain distance. But the drug was not proved to be safe and effective at the same time. It is worth mentioning that the drug has been conditionally approved in Europe.