We’ve entered a new chapter for families with Duchenne, and 2017 has been a year of incredible promise.
Yes, we’ve faced some setbacks, but we’ve also seen incredible successes, like recent breakthroughs in gene therapy. It’s essential that we keep fighting, with the same commitment to innovation that’s always guided us, and as we expand PPMD’s Gene Therapy Initiative, we have an opportunity to do just that.
Our fight has always been a team effort, and I’m calling on you today to help advance our Gene Therapy Initiative, so that we can continue to support revolutionary research in approaches like microdystrophin and CRISPR, and begin exploration of potential strategies like GALGT2.
One thing I’ve learned throughout my career is that breakthroughs don’t always come when we expect them to. More often than not, giant steps forward are the result of seemingly incremental advances.
This is a big moment for gene therapy, even outside of the Duchenne community. The FDA’s recent approval of Kymriah (a gene therapy that treats an aggressive type of leukemia) and Dr. Jerry Mendell’s recent success with gene therapy in SMA are great examples of why it’s one of the most promising therapeutic approaches out there. And why we have to keep going.
Thanks to your commitment, PPMD has already started driving this technology forward, with our exploration and investment in microdystrophin, CRISPR, and now GALGT2. Our Gene Therapy Initiative looks closely at each potential therapy and maximizes the opportunities each presents. It’s part of PPMD’s commitment to all people with Duchenne. Donate today to make this breakthrough research possible.
Thank you for supporting PPMD this holiday season.
Senior Vice President, Research Strategy
Parent Project Muscular Dystrophy
P.S. With so much momentum in gene therapy, now is the time to expand PPMD’s Gene Therapy Initiative so that we can take a deeper dive. Every dollar you give makes further exploration and expansion possible and brings us closer to fulfilling our mission. Have your donation doubled today to advance this potentially game-changing research.