Six years ago when our son was diagnosed with Duchenne Muscular Dystrophy several doctors told us there would be as close to a cure as possible in two years. Obviously they were being optimistic. At that time, research on Duchenne had been in place well over 20 years.
Today well over 26 years have passed by with a plethora of scientific data on Duchenne. And today, my son loses muscle every second of every day. Simply, none of that research has come to the market. One year ago, the promise of Sarepta's Eteplirsen and the seemingly positive attitude you, the FDA had towards this therapy had me praising the Lord for the hope of my son's muscles surviving. This felt nothing short of a miracle. Then the reins were pulled when GSK's product failed. And again, the fight, the battle, the plea, the prayers to move Eteplirsen forward continues as still, my son struggles to even lift a fork to his mouth at dinner. He is ten years old.
We need you, the FDA, to realize parents of son's with Duchenne have nothing but the face a robber who is stealing our sons every second of every day. And each day you wait, you kill them faster. We can't wait another 26 years for a therapy. Though I am not a scientist, doctor or even qualified to tell you the details of this drug I can tell you it works on these boys. I can tell you that I want this drug for my son. He is missing exon 49 and 50, clearly he would benefit. It will help him as right now there is no help no hope just watching as he struggles daily with this robber who has one goal destroy this precious child.
Thank you for hearing our plea.
Jen Dumm John Owen Dumm's Mother 3.7.2014