A note from Debra

The Wait for An Approved Drug for Duchenne Continues

Posted on May 25, 2016 1:57 pm

 

The wait continues.  Sarepta Therapeutics announced this morning that the FDA will not complete the review of the eteplirsen new drug application by the PDUFA date.  Considering the thousands of lives that could be affected by a decision on eteplirsen, we understand and appreciate the care the FDA is taking in its review of this innovative drug. We are hopeful that the delay is a positive sign.  Today, the Duchenne community needs to remember that a delay is not a no.

CureDuchenne provided early funding to Sarepta for the development of eteplirsen, and we have worked together to reach this point in the years since. While we can certainly wait for the FDA’s decision, we cannot afford to lose another generation of boys to Duchenne because there weren’t treatment options. It is urgent that we get the right combination of approved treatments for our boys.

Every delay is taking precious time away from our boys. Time is not on the side of those with Duchenne.  We must enjoy every moment with our children and every hug.  Watch a video about the Procko family who’s son Evan, 12, is on the Sarepta trial is confronting the disease and living in the moment.

We continue to remain hopeful that we will have an approved treatment for all those with Duchenne soon.

 

NORD Rare Impact Awards

Posted on May 18, 20165:13 pm

  NORD hosted the 2016 Rare Impact Awards to bring together the rare disease community and to honor and celebrate outstanding achievements.  Last night, Debra Miller, founder of CureDuchenne, was one of the esteemed honorees who was recognized for moving us forward in the fight against rare diseases.  Congratulations Debra for your tireless effort to Read more »

Happy Mother’s Day

Posted on May 8, 20164:55 pm

  From one mom to another, I wish you a Happy Mother’s Day from the bottom of my heart. As I think about the most wonderful gift in my life, my son Hawken, I am filled with an all-consuming joy. His humor, laughter and innate kindness surprises and encourages me every day. And that’s what Read more »