My two oldest children, Max and Austin, have a lethal genetic disease called Duchenne Muscular Dystrophy (DMD). Right now, only one of my sons has access to a new "miracle drug,” and it has changed our lives. I am watching one of my boys thrive, as my other gets tragically worse while he waits for access to the medication that could save him.
Every day, children with DMD get weaker, it causes loss of muscle, to the point where children with the disease cannot breathe on their own. It is a slow death sentence.
Miraculously, one of my sons has been saved. Max, just 10 years old, was lucky enough to take part in a medical trial. For the last 60 weeks, Max has been taking this new medication. This year, my son who was once dependent on a wheelchair most of the day, marched in a Halloween parade.
But our journey has been bittersweet. As we watch Max get better, we also watch his older brother, Austin, 13, get worse. He suffers, silently, as his disease progresses. Austin was unable to take part in the drug trial that helped his brother so much.
Although Austin doesn't walk, he still has strength in his upper body, which this drug could help him keep. It could save him from spinal surgery, a tracheostomy, a feeding tube. It could allow him to drink and eat. It could keep him sitting up. It could allow him to hug his family.
Eteplirsen is the first hope we have had since Austin and Max were diagnosed with Duchenne. It has the ability to stop this disease in its tracks...and the boys who need this drug simply don't have one more day to waste, waiting for it. Austin does not have one more day to waste, waiting for it.
This development comes at a very important time. A law was just passed, allowing the FDA to move drugs like these, for fatal illnesses, like Duchenne, through an accelerated approval process.
Thousands of boys in the United States are suffering with DMD. If the FDA allows American boys access to this drug, it will save thousands of lives -- and could help many more across the globe get the care they need.
Please, Austin and Max both deserve a chance to thrive. Tell the FDA to make this important drug available to all children with Duchenne Muscular Dystrophy.