Update: Focusing Forward

Dear Friends,
As we set out on the New Year, we’re looking back on what our community has accomplished and focusing forward on how to make more progress.

2016 was a monumental year, with the first-ever drug approval for Duchenne. We are proud to have been a part of that and grateful to everyone who helped make it happen. But we know there is still much to do. Eighty-seven percent of Duchenne patients, including our own boys, Joseph and Charley, will not be helped by Exondys 51 (formerly eteplirsen). Although the countdown to the New Year has come and gone, the clock still ticks loudly every single day for our Duchenne community, and that keeps us up at night.

When we look back at what it took to get the first treatment approved—the herculean effort of time, money, people, and energy—we know that we cannot proceed down the same path each time a drug company faces resistance or hits a bump in the road. The questions driving us are: How do we make an impact on the bigger picture, the regulatory process for Duchenne drugs? How do we smooth the path for all treatments to come?

We want you to know that, although we are quieter now, we are working diligently to bring about change. We are taking stock, thinking strategically, and tapping into the brightest minds to plan next steps. We will be in touch with updates.

Thank you for staying the course.

Marissa Penrod & Tracy Seckler
Co-Founders, the Race to Yes