I am a 22 year old living with Duchenne, and opportunities for clinical trial participation are few and far between. It is one of the frustrating realities of my diagnosis and my age. Although it has never swayed my commitment to this community, I have to be honest. It can be discouraging at times.
But gene therapy is likely to be a game changer for adults like me living with Duchenne! Since my diagnosis in 1998 gene therapy has seemed to be a far off opportunity, and now we are so close to that reality. And Parent Project Muscular Dystrophy is exploring everything gene therapy technology has to offer with their Gene Therapy Initiative.
For only the next two days, every dollar you give will be matched until we hit our goal of $500,000. All donations support PPMD’s Gene Therapy Initiative, including research in microdystrophin, CRISPR, GALGT2, and future gene therapy technology. Please give today.
I’m a proud member of Parent Project Muscular Dystrophy’s Adult Advisory Committee (PAAC). Through this incredible group of peers, I get to play a role in improving the landscape of Duchenne research and advocacy for years to come. I’m helping to lead the navigation of my own care and, despite some of the physical limitations of Duchenne, I have never felt more independent!
Gene therapy holds promise for people like me—adults living with Duchenne. And PPMD is putting its weight behind gene therapy like never before.
I truly believe that we are closer than ever in our fight to end Duchenne. Support PPMD’s Gene Therapy Initiative. Only two days left to have your gift matched! Not only will your donation be doubled—it will be used to bring promising treatments to people like me even sooner!
I’m so proud that PPMD remains actively supportive of the adults with Duchenne whose families they’ve supported from the beginning. Words cannot express how truly thankful I am to be on this amazing “journey” with Pat and everyone at PPMD, as well as you and the rest of this inspiring and courageous community.
Happy holidays from my family to yours!
PPMD's Adult Advisory Committee
P.S. Don’t forget: all donations made between now and the end of the year will be matched! In other words, your gift is doubled. With only two days left for the matching gift, now is the time to give—now is the time to join the fight to end Duchenne!