In 1992, I was diagnosed with Duchenne at two years old, significantly earlier than the typical diagnosis age of 6 or 7 at that time, due to a family history. In the 90’s, our understanding of Duchenne – and this community of people living with it – looked a lot different.

But even then, 30 years ago, PPMD worked tirelessly to connect families with the best physicians; to invest in groundbreaking research that would make meaningful change; to end Duchenne. As Pat Furlong has said, gene therapy was just a whisper then, the safety risks were too great. Look how far we’ve come.

Advocating for people with Duchenne has been my lifelong passion. Over the past 25 years, I‘ve emphasized that those with Duchenne can lead fulfilling lives. For me, research was more of an afterthought; gene therapy seemed a far-off possibility.

However, this past May, at the age of 34, I found myself serving as the patient representative on the FDA Advisory Committee that would recommend accelerated approval of the first gene therapy for Duchenne.

This is why I am proud to announce that thanks to the generosity of our community, every donation to support PPMD’s Gene Therapy Initiative will be MATCHED through 12/31.

Your gift to support PPMD’s Gene Therapy Initiative will be DOUBLED through midnight on 12/31.

Donate

As of now, it’s unclear as to whether I will ever benefit from these treatments, but I have seen the amazing things gene therapy can do for patients and their families. I stand by what I said at the Advisory Committee meeting in May — I am determined to make sure we don’t lose another generation to waiting.

Just like PPMD, I am committed to fighting for every future. Please join me in this fight with your gift today!

We have reached a remarkable milestone with the first approved gene therapy, but it’s just that: the first. We have more questions to answer, more futures to fight for. With your support, PPMD will fund additional research to overcome the numerous challenges we still face in the rapidly growing field of gene therapy.

PPMD’s goal this holiday season is to raise $500,000 for its Gene Therapy Initiative, which 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, seek out solutions so that pre-existing antibodies are not a barrier to otherwise eligible individuals, and accelerate the path to repeat dosing.

I ask you to join me in supporting PPMD’s Gene Therapy Initiative. Give today and your donation will go twice as far.

Even though gene therapy isn’t an option for me right now, I am very grateful to be where I am today, at this particular moment in time, because I can make a difference for the countless individuals living with Duchenne, now and in the future. No doubt the future holds great new wonders. Let’s come together and move this research forward.

Double your gift today. Together, we will fight for every future.

Donate

Thank you,

Buddy Cassidy
PPMD Advocate

P.S. Your gift goes twice as far now through 12/31. Fight for every future with a donation today.