My son Connor’s diagnosis of Duchenne turned my family’s life upside down. In order to prioritize Connor’s comfort and quality of life we moved from Chicago to Florida. We’ve changed jobs, schools, and care teams, rearranging nearly every aspect of our lives. Living with Duchenne and being part of the PPMD community has taught us to embrace change when it comes and to work tirelessly to create change where it’s needed.
After being told by doctors that he was a great candidate for a gene therapy study, Connor was two weeks away from his fourth birthday when screening revealed he had pre-existing antibodies. We were so hopeful that this particular gene therapy would be an option for Connor, and an effective one, but that, too, suddenly changed.
PPMD’s Gene Therapy Initiative aims to broaden access to these treatments by working to overcome barriers like pre-existing antibodies for otherwise eligible individuals like Connor and so many others. And I am hopeful that day will soon come. That is why I am urging you to join me as I fight for every future by supporting PPMD’s Gene Therapy Initiative with a gift today. Until midnight on December 31st, your gift will be DOUBLED thanks to the generosity of our community.
As a community, we have driven forward so much progress in the world of Duchenne. We have reached a remarkable milestone with the first gene therapy, but our work is not done. Gene therapy is not an option for Connor and many others right now, but I am hopeful that can be changed in the near future.
Your gift to support PPMD’s Gene Therapy Initiative goes twice as far now through 12/31. Fight for every future with a donation today.
DonateBeyond addressing antibodies, PPMD’s Gene Therapy Initiative also aims to help us deepen our understanding of the long-term effects of this treatment on the heart, prioritize research for next-generation and future-generation gene therapies and combination therapies, and accelerate the path to repeat dosing. These goals will help us reach a future where gene therapy can become a real possibility for everyone who needs it.
When I think about the positive changes this community has made in the fight to end Duchenne since Connor was diagnosed, I am hopeful. With each dollar you contribute, you are giving hope to our entire community.
Double your gift today and together, we will fight for every future.
DonateThank you,
Vishva Desai
P.S. Your gift goes twice as far now through 12/31. Fight for every future with a donation today.