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

Exondys 51 vs. Ataluren

This drug was called Exondys 51 and allowed genetic machinery skip over a specific mutation which has been proved to be the same mutation 13% of people with Duchenne have. Nevertheless, it was not the case of the Vertin brothers, and they were not helped by the drug at all. So, they had to try Ataluren. This drug forces cells to produce proteins instead of a mutation of this.

Thanks to the clinical trial they are on, they are able to get their medication for free as well as the expenses for those trips from Nebraska to Washington, D.C. The way ataluren works is by ordering a cell to use something different to build the protein.