Dear NUCDF Community,
One thing became abundantly clear during this year's NUCDF Family Conference: our community is shaping the future of UCD care together.
Looking around the room and seeing so many children, teens, and young adults living with urea cycle disorders was incredibly moving. Today, we're celebrating young adults pursuing college, building careers, living independently, and creating futures that once seemed unimaginable.
That is hope.
During my opening remarks, I shared five reflections inspired by what we've heard from families over the past two years. Whether you joined us in Memphis or are following from home, these reflections belong to our entire community.
1. We seek trusted information.
Families want accurate, balanced, and timely information to help them make informed decisions about treatments, clinical trials, and everyday care.
2. We are navigating more choices than ever before.
New therapies and advances are creating tremendous hope and new questions. NUCDF is committed to helping families navigate this evolving landscape with confidence.
3. Living with a UCD is a lifelong journey.
From diagnosis through adulthood, every stage brings new challenges and opportunities. We're committed to supporting families every step of the way.
4. Our voices are shaping the future.
Patients and families are no longer simply participating in research, they're helping guide it. Lived experience is essential to advancing meaningful, patient-centered care.
5. Hope lives here.
Hope is found in scientific breakthroughs, in the resilience of our families, and in a community that continues to lift one another up.
Those reflections came to life throughout our conference. One of the most exciting milestones we celebrated was the announcement that Dr. Rebecca Ahrens-Nicklas and her team at Children's Hospital of Philadelphia received a five-year, up to $38.9 million ARPA-H award to advance personalized gene-editing therapies for rare genetic diseases. NUCDF is honored to serve as a partner in this groundbreaking initiative, helping ensure that patient and family perspectives remain central as these therapies move toward the clinic.
We also shared our partnership with Case Western Reserve University on the NIH-funded CONNECT initiative, giving our community the opportunity to help shape conversations around the ethics, access, and future of emerging therapies. These collaborations represent exactly what NUCDF has always been about: bringing the patient voice to the table when decisions are being made.
For nearly 40 years, NUCDF has connected families, researchers, clinicians, and advocates around one shared belief: families come first.
Our conference theme this year was Bridge to the Future. When I think about that bridge, I see every family whose courage, advocacy, and lived experience have helped build it.
The future of UCD care will be built together—through scientific discovery, lived experience, and a community that never stops believing in what's possible.
Thank you for being part of that future.
