Race to Yes
The Race To Yes mobilizes people who support rapid regulatory action to advance safe and effective treatments for Duchenne muscular dystrophy and amplifies their collective voice as a mechanism for change.
We represent people who believe the participants in the American regulatory system—the FDA, the drug developers, the President, and Congress—have an obligation to urgently and proactively pursue ways to speed safe and effective treatments to patients. As research advances, new opportunities require new, nimble, thinking about how to work together to deliver treatments as efficiently and effectively as possible to patients with rare, fatal diseases like Duchenne. The Race to Yes is the people’s movement to make this a priority. Science is difficult enough—our system shouldn’t get in its way.
Founded in 2013 by Charley’s Fund and Team Joseph, The Race to Yes pursues a three-pronged approach to achieving this end: direct conversations with the FDA, engaging elected officials, and public awareness/activism.
To learn more and join the movement, go to theracetoyes.org
The Race to Yes The Race to Yes
The Race to Yes: A Chance to Improve the Process
by carolinn on September 21, 2017 at 4:53 pm
Next Thursday, an FDA advisory committee will meet to consider whether to approve a treatment for Duchenne muscular dystrophy. The last time an FDA advisory committee met to review a therapy for this fatal childhood disease, things did not go smoothly. — Portrayed in the media as a heated showdown between the FDA and desperate... Read more » […]
Update: Focusing Forward
by coreygraham on January 24, 2017 at 11:08 am
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... Read more » […]
Breaking News: Eteplirsen was Approved
by coreygraham on September 19, 2016 at 7:49 pm
Today, the first safe and effective treatment for Duchenne muscular dystrophy—Eteplirsen—was approved. This is a proud day for every member of our Duchenne Community who worked for this moment and this historic victory—the experts who weighed in with the FDA, the community who rallied, and most of all the 12 boys in this trial who... Read more » […]
FDA delays decision on eteplirsen
by carolinn on May 25, 2016 at 9:29 pm
Sarepta Therapeutics announced this morning that the FDA has indicated it will not reach a final decision on eteplirsen by the May 26th deadline. We understand and share the frustration many of you feel that the FDA still hasn’t made a final decision on eteplirsen despite strong scientific evidence that supports approval. We have already... Read more » […]
The Next Lap in the Race to Yes
by carolinn on May 19, 2016 at 3:07 pm
By next Thursday, the FDA will make a decision on approval for eteplirsen. Whether the FDA’s answer on eteplirsen is “YES,”or “YES, BUT…,” or “NO,” the race is far from over. If eteplirsen is approved, we will celebrate this historic milestone of the first ever therapy for DMD. For the 87% who cannot benefit from... Read more » […]