So much has changed in the Duchenne research landscape since we were founded back in 1994, but your commitment has been a constant since our story began.
You’ve never stopped fighting, and the promise for our community has never been greater, with recent breakthroughs in gene therapy, one of the most exciting approaches we’ve ever seen. Today, we have an opportunity to further explore this vital work through PPMD’s Gene Therapy Initiative and increase our investment in the approaches being pursued: microdystrophin, CRISPR, and GALGT2 technologies.
PPMD is raising $400,000 to expand our Gene Therapy Initiative—our biggest holiday goal to date! Over the last 12 months, gene therapy technology has dominated the headlines, and PPMD is proud to have made early investments to explore each promising pathway. By supporting the expansion of our Gene Therapy Initiative, you are helping PPMD fill critical gaps in the development pipeline so that these therapies can maintain their momentum.
And, thanks to an extremely generous family in our community, every donation will be matched dollar for dollar until we reach our goal. Inspired by the potential of PPMD’s Gene Therapy Initiative to help all people living with Duchenne, this family will double your donation today. Please give now!
Our recent victories are a testament to what we’re capable of when we come together as a community. But as proud as we are of everything we’ve accomplished, we won’t rest until every family can turn to a treatment to end Duchenne. We believe that the expansion of gene therapy is critical to that fight.
So far, our Gene Therapy Initiative has focused largely on dystrophin replacement using a microdystrophin and the possibility of CRISPR technology to treat Duchenne. GALGT2 is another strategy with game-changing possibilities, including the potential to treat the entire Duchenne community.
Your commitment got us this far, and now, as we set out to build on our incredible momentum, we’re asking for your courage to keep fighting. Every gift takes us closer to our goal. Make a tax-deductible donation today to have your gift doubled.
This is about advancing research forward by exploring gene therapy, the most promising approach available today, from every angle. It’s about fighting for all people with Duchenne—like we always have, and like we always will.
P.S. Expanding our Gene Therapy Initiative is vital to our mission of finding a solution for every single person affected by Duchenne. Please help us raise the $400,000 necessary to advance these revolutionary approaches. And remember: your donation will be doubled through the end of the year!