First Drug Approved for Duchenne: A Beacon of Hope for the Duchenne Community

My husband Paul and I have dreamed of this day since our son Hawken was diagnosed with Duchenne almost 15 years ago! We are overwhelmed with joy to announce that the FDA has officially granted accelerated approval for Eteplirsen for the treatment of Duchenne Muscular Dystrophy. This is the first approved drug-therapy and a major breakthrough in the fight against Duchenne.

We couldn’t be prouder of the researchers at Sarepta Therapeutics. Our heartfelt appreciation for all the Duchenne families who have participated in this and other clinical trials to benefit the entire Duchenne community.

I am certain that many of you experienced the same devastating heart drop that we did when you were told that your son would be powerless as this disease ravaged his young body. While we are united in that pain, we are also united in our fighter’s spirit. Paul and I were not ready to let our son die without battling for a glimmer hope, and we know you feel the same way.

CureDuchenne provided early funding for Sarepta Therapeutics, the biopharmaceutical company that developed the exon skipping treatment behind Eteplirsen. This funding, led by Eric Hoffman at Children’s National Medical Center and also supported by the Foundation to Eradicate Duchenne,allowed the company to move into human clinical trials and continue to move forward with the development of Eteplirsen.  We are proud to have supported Sarepta early in the development of this drug.  The risks of success at that time were high but we agreed if there was a chance for a positive treatment, it would be worth taking. We are grateful for our donors and supporters who helped fund this research.   The risk has paid off for the boys who have the specific genetic mutation that Eteplirsen targets. Unfortunately, our son Hawken isn’t one of them.

This scientific leap and FDA approval is just the stepping off point, though. Thanks to modern technologies, the brilliant researchers at Sarepta Therapeutics and all of the support from each of you, we know more about the genetic mutation that causes Duchenne than we ever have. The success of Eteplirsen is a building block for future treatments. However, Eteplirsen is effective for only 13 percent of those with Duchenne. While this is a huge therapeutic discovery, 13 percent is still not enough. Every person with Duchenne deserves hope, a future and a cure. All of us at CureDuchenne will continue to fight and will not stop until we reach that goal of a cure for every person with Duchenne.

Thank you to Edward Kaye and the entire team at Sarepta for developing this drug and your commitment to the Duchenne community.  Thank you to the families who participated in the trials. Thank you to the FDA for approving this first Duchenne treatment. We hope this is the first of many approved treatments for Duchenne.

Celebrate with your loved ones, and remember that all of this is just the beginning. Thank you for your support and joining us in this fight!