1. Because Duchenne is the leading genetic killer of children – it has a 100 percent fatality rate.
2. Because there is a drug that has been proven to be safe and effective, eteplirsen, that could potentially save thousands of lives, but it needs FDA approval.
3. Because over 100,000 people spoke and petitioned the White House to ask the FDA to grant accelerated approval for safe, effective treatments.
4. Because this pending FDA decision could determine whether this generation of children is the last to die from Duchenne or the first generation to live.
5. Because leading scientists and clinicians all say Yes. These are just a few who do: