As a community, we must continue to demand that the FDA use all available tools given to them by Congress to speed the approval of new therapies to all who so desperately need them. The voices of patients and caregivers are critical to the process of drug development and we believe the message is being heard.
Here are 3 simple actions you can take to support & amplify that message:
- Urge your Representative to sign the Duchenne Congressional Funding Letter
Our funding letter currently being circulated by Representatives Peter King and Doris Matsui includes important provisions through report language to focus the agencies (FDA, NIH, CDC) working on Duchenne. Within the letter, we urge the FDA to use every tool at its disposable to speed the approval of new therapies for Duchenne.
Ask your member of Congress to sign the Duchenne funding letter and support this message.
- Sign the petition
A petition is currently circulating on WhiteHouse.gov to urge the FDA to use the accelerated approval pathway (laid out/encouraged by FDASIA, a bill we worked tirelessly with others in the rare disease community to pass) for approval and access to safe, effective therapies for Duchenne. If our community reaches 100,000 signatures by March 29th, we will receive the President’s attention.
We are currently just under 50,000 signatures. Sign the petition today!
- Share your story with the FDA
If you had a chance to talk to the FDA, what would you want them to know? How does Duchenne affect your son, your family, your life? What are you hopes and dreams for the future? We will compile your stories and share them with the FDA so they have a better understanding of Duchenne when making decisions.
Here are some specific things that the FDA would like to know your opinion about:
- The severity of Duchenne and the impact on your life
- The symptoms of Duchenne that matter most to you
- Your perspective on the available therapies/interventions or lack there of
Share your story with the FDA so they have a better understanding of Duchenne. You can also view current stories that have been submitted.
Share these actions with everyone in your network! Facebook, Twitter, etc. links are below.
Together, the FDA will hear our message: Speed the approval of new therapies to all who so desperately need them.