What is the mission of the Race to Yes (R2Y)?
The mission of R2Y is to accelerate the Food and Drug Administration’s (FDA) approval of therapies to treat Duchenne muscular dystrophy in time to benefit this generation of children living with Duchenne.
When and why was the Race to Yes started?
The Race to Yes was launched in late 2013 after the Food and Drug Administration (FDA) reversed course on accepting a New Drug Application (NDA) for eteplirsen, a promising new treatment that works by producing dystrophin, the protein that kids with Duchenne are missing. Well before the Race to Yes was created, several nonprofit organizations, numerous world-renowned scientists, and individual families had been running this race on their own. As a direct result of their efforts, the FDA indicated it would accept an NDA for eteplirsen in June 2013. But by the end of 2013, the FDA reversed its willingness to accept an NDA, a decision that was based misinformation and confusion. The Race to Yes was launched.
The Race To Yes was created to get us back on track and leverage all the incredible work that had already been done, to mobilize even more people who support rapid regulatory action to advance safe and effective treatments for Duchenne, and amplify our collective voice as a mechanism for change.
Does R2Y support the speedy approval of all therapies for Duchenne, or just eteplirsen?
R2Y’s goal is to get the FDA to act expeditiously on ALL safe + effective candidate therapies for Duchenne. Our current focus on eteplirsen is because the FDA—not the science or the data on safety or efficacy—is the barrier to swift review and approval. When there are other therapies before the FDA that need our help to cross the finish line, we will support those therapies as well. The Race to Yes is aimed at getting the FDA to act with the same urgency for Duchenne that it has for childhood cancers, HIV, and so many other conditions. The FDA should use each and every tool Congress has given them to expedite therapies for rare, fatal childhood diseases.
The good news is that we finally have many candidate therapies in the pipeline. The challenge is whether the FDA will act on them quickly enough to benefit our kids. In the case of eteplirsen, the FDA’s actions to date have delayed and derailed efforts to get this therapy to kids who can benefit. And that delay does not just affect kids who can benefit from eteplirsen. It affects those counting on the follow-on therapies to skip other exons. Equally important, the way the FDA handles the approval of eteplirsen could have important implications for ALL Duchenne therapies, including those beyond exon skipping.
Is R2Y sponsored by or connected to any drug companies?
What nonprofit foundations support R2Y?
R2Y is paid for directly by Charley’s Fund (CharleysFund.org) and Team Joseph (TeamJoseph.info). While neither Charley nor Joseph can benefit from eteplirsen, we all have a shared mission to serve this generation of children—our generation of children—who are waiting for safe and effective treatments.
Many other organizations and over 106,000 families, friends, citizens, and celebrities have joined the Race to Yes movement by raising their voices, signing petitions, directly contacting their elected leaders and FDA officials, and talking to the media.
How do I get updates from R2Y?
Can I support R2Y and other organizations that are funding medical research and other important projects?
R2Y does not accept donations but we encourage you to give generously to one of the many impactful nonprofit organizations that directly fund and support Duchenne research.