We’ve been working toward this moment for years. And the time for action is now.
When I started with PPMD in 2009, Pat Furlong and I talked a lot about what it would take to end Duchenne. We knew it wouldn’t be one thing, and we knew it wouldn’t be easy. We believed that only a combination of therapies could address the entire disease within the body.
And now we are seeing more progress than ever with a variety of drugs making their way through the clinical trial process, some of which plan to file new drug applications in 2015. The possibility of combination therapeutics is—right now—closer to becoming a reality.
Early next year, we plan to fund a group of research projects that, together, we believe will help us take a big step forward in ending Duchenne. We need to raise $400,000 before year-end to make that funding possible. The time is now to push that progress forward in a big way. Donate today and your gift will be doubled by a generous match!
We’re energized by the promise of using multiple drugs in combination to fight Duchenne. A drug that increases muscle mass could be combined with a drug that stimulates the production of dystrophin or utrophin, first building muscle mass and then stabilizing that new muscle. Or a drug that decreases inflammation could be combined with one that reduces or reverses fibrosis in the muscles.
We may ultimately wish to use all four approaches together. But for now, we need to determine if these drugs can be used together safely, and if so, whether the combination produces a greater effect than any drug alone.
The foundations Cure Dale’s Duchenne and Rally For Ryan share our great optimism that this combination approach is the way forward, and they’ve generously agreed to match all online donations from now until the end of the year. We’ve set an ambitious goal of $400,000, and we need you to donate now to help us take full advantage of their generosity.
As Pat mentioned in her email a couple of weeks ago, we need to prepare now for the next stage of Duchenne research by systematically trying drugs together, first in animal studies and then in human trials. We also need to understand how regulators will view combination therapies and lay the groundwork now for rapid testing and approval of combinations.
We believe that most of these therapies will be much stronger when they work together. The same is true for our great community—there’s nothing we can’t do when we come together to end Duchenne.
I hope that if you believe in innovation and the strength of combining approaches in the fight to end Duchenne, that you will join me and my family by making a donation to PPMD.
Enjoy the holidays with your family and thank you for supporting PPMD!
P.S. Did you miss our webinar yesterday? Click here to download the full presentation and learn more about PPMD's combination therapies initiative.