Autsin Leclaire and his brother Max suffer from Duchenne muscular dystrophy. It is a rare muscle-deteriorating disease with no recovery. Max qualified for a blind test run of eteplirsen, but unfortunately his brother did not.
The family saw immediate improvement in Max and knew, since patients with this disease don't get better, he must be on the drug. They petitioned the company to release the drug to their son Austin as well. However, while Austin continues to deteriorate, Sarepta says, due to complex financial, regulatory, political and manufacturing roadblocks, Austin is ineligible. The condition is life-threatening. According to Max and Austin's mom, "We can't have one son who has had his life saved and is the first kid to survive this disease, while we have our other kid at home who is going to be the last kid to die from it."
It is important that Sarepta and the FDA understand that this can not happen, that they can not allow one brother to live, while his family watches the other lose his battle.