As a mother who lost two sons to this dreadful disease, I am driven to find every way possible to help bring new therapies to families, to end Duchenne. PPMD supports and agrees with all parents urging the agency to accept Sarepta’s submission for the approval of eteplirsen, as well as any other therapy that shows similar promise. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.
Sarepta’s 120 week stability data on both the six minute walk test, as well as, pulmonary function is good news for the Duchenne community. For the 12 young men in the study, we are thrilled. And for those waiting, we see hope on the horizon. We congratulate Sarepta for their continued commitment to people with Duchenne and we hope the FDA…Continue
Patrick was 8 years old. Steroids were not recommended. There was no imaging and no way to predict his loss of ambulation. Sure, he fell occasionally. Some days more than others. There was one day in the summer of 1988. We had been active most of the day. We went swimming, to the mall, and ended the day with a cookout. Patrick walked and walked. He asked me to help him to the bathroom. He fell. It was one of those FALLS, as if his legs were pulled out from under him. I had no excuses in my…Continue
12/12/13 8:30 AM
There are days when the stars align just right. Seems to me, December 12, 2013 was one of those days.
Eighteen members of the FDA arrived and were seated around a U-shaped table near the front of the…Continue
For our families, Duchenne research can’t move fast enough.
There is no lack of ideas or interest—7 public companies and more than 15 biotechs are focused on finding treatments for Duchenne. But promise is only one part of the story. We need to actually change the landscape and bring drugs to market faster.
You can help make it happen with one simple action:…Continue