Dear Friends,
Naomi, age 4, has A-T.
We’re feeling so optimistic about the new year, given the accelerated progress we’re seeing in our search for therapies—and ultimately, a cure—for ataxia-telangiectasia (A-T)!
A few weeks ago, we shared exciting updates from the Gene Therapy for A-T workshop we recently held in Boston. The event brought together leaders who have brilliant minds as well as track records of getting things done, sparking collaborations that are already gaining momentum. And yet, while gene therapy is an extremely promising avenue, it’s just one of many areas where we’re seeing remarkable advances. Here are some others:
- Repurposing existing drugs to accelerate the path toward treatments.
- Leveraging cutting-edge technology to unlock new possibilities.
- Exploring new biological targets and mechanisms that may lead to drugs addressing neuroinflammation and mitochondrial dysfunction in A-T.
- Partnering with pharmaceutical and academic teams to move clinical trials forward.
- Exploring innovative brain scanning techniques to uncover treatment clues.
- Expanding our Global A-T Family Data Platform by collecting and analyzing blood for biomarker studies, saliva for whole-genome sequencing, and other types of data – all of which enable new research efforts worldwide.
We truly believe 2025 will be a year of transformative progress, bringing us closer to therapies that can benefit people with A-T of all ages. None of this would be possible without the tireless efforts of A-T families and friends, who raise critical funds year-round to drive these initiatives.
If you haven’t yet had a chance to make a year-end donation, there’s still time to join us in advancing this important work. Your support will have a direct impact on bringing hope and meaningful treatments to children and young adults living with A-T. You can make a gift here.
Thank you for being such an important part of this journey. We’ll keep you updated in the months ahead, but for now, we wish you and your loved ones a safe and joyful start to the New Year!
All the best,
Jennifer Thornton, Executive Director