Duchenne is getting a great deal of attention these days. With the excitement around the Sarepta trial and more trials coming online, more attention to Duchenne is a great thing as we continue to raise awareness. Personal stories about families only help to educate the public on Duchenne. It allows them to relate and perhaps to think, If this was my son, what would I do??
A recent petition on change.org started by parent Jen Mcnary has gained a great deal of traction reaching almost 5000 signatures in just a few short weeks!
This petition, along with our ongoing efforts in Washington including meetings with the FDA and the upcoming reauthorization of the MD-CARE Act, says to the powers that be in D.C. that the voice of this community is united, strong, and important. The FDA continues to hear from various people and groups, through various formats and outlets, that Duchenne matters to our community and to all those who care for our children.
Over the past year, PPMD has worked tirelessly on a critical piece of legislation, called the Food and Drug Administration Safety and Innovation Act (FDASIA) or PDUFA V, ensuring that the most important rare disease provisions were included in the final legislation, including one on accelerated approval. We are hopeful that as drugs like eteplirsen come forward, the FDA will be responsive to this legislation and utilize all the tools in its arsenal to make thoughtful, yet expedited decisions on therapies.
Awareness continues to be one of our greatest challenges and most important weapons in the fight to end Duchenne. We are always looking at ways to bring the story of Duchenne into the main stream. Whether it’s reaching out to major media outlets to take a critical look at clinical trials, or our upcoming survey that will provide detailed descriptions of the Duchenne experience to the FDA, we need to keep shining a spotlight on Duchenne and to raise the public consciousness about the disorder and how it affects families.
We will continue to do whatever it takes to educate the public, industry, and regulators about Duchenne. Efforts like Jenn Mcnary’s petition help give a face to Duchenne. At a time when there is so much noise and so many distractions in our hectic lives, it is great to see a story move so many people and where our collective voice can say to the world, Duchenne matters. We want the FDA to know how much it matters to our community and to all those who care about our sons (and some daughters). No one can do everything to end Duchenne, but we can all do something. .