As many of you know, after gathering more than 106,000 signatures and then waiting over four months, last night we received a response to our White House petition asking for accelerated approval for safe, effective treatments for Duchenne.
We are glad that at last, the White House is publicly acknowledging innovative pathways are needed and appropriate to use to speed approval for treatments for Duchenne. We couldn’t agree more. But our kids still don’t have access to the treatments. It’s time to run the next lap in this race. The starting gun has fired, the lane is open, the path is clear. Now it’s time for the drug companies and the FDA to RUN – run faster and run harder than ever before.
For every child with Duchenne, time is muscle. There is a very limited window of time when our children still have enough muscle for these therapies to help. Every day, our children lose that muscle, and the window closes a little bit more for this generation of children with Duchenne. That’s why the Race to Yes mission is so important and so urgent. The FDA and the drug companies must give our children access to these treatments without unnecessary, painful placebo trials. They must have frequent, in-depth communication and collaboration to make sure the process is moving as quickly as possible.
To get a response like this from the White House and FDA – addressing a specific disease, company, and treatment – is rare. We reached this point because of YOU and YOUR EFFORTS. Thank you for everything you have done. We won’t stop until our kids get the safe, effective treatments they deserve.
Tracy Seckler & Marissa Penrod
On behalf of The Race to Yes