Celebrating 3 Years of Hope
Dear Channeling Hope Community,
This is one of the most exciting moments the rare disease community has ever seen! Advances that felt like science fiction just a few years ago–gene therapies, AI-accelerated drug discovery, and treatments designed for a single patient–are moving from the lab toward the clinic at a remarkable pace. As we celebrate three years of the Channeling Hope Foundation, we are proud of how far we have come together: from a small group of families searching for answers to a global community of patients, scientists, and clinicians driving NALCN research forward. Advocacy can be daunting, the science is complex, and the road to treatment feels long, but together we are making progress.
WHERE WE’VE COME
As we celebrated our second anniversary in 2025, we committed to our research priorities being guided by the perspectives and priorities of patients and caregivers. To put this into action, we convened a joint family–scientist conference that brought together caregivers, clinicians, and researchers to identify shared clinical and research priorities. Importantly, we launched a three-question Patient Priority Survey to better understand our community’s most urgent concerns, alongside a more comprehensive Patient-Oriented Outcomes Survey designed to identify the clinical trial endpoints that matter most to families and caregivers. We also hired a full-time Research & Operations Manager to help engage our community and empower us all to be active partners in NALCN research.
WHAT’S NEXT
Our horizon is bright. Novel AI models and cells derived from patients in our community have identified promising drug repurposing targets and initiated pipelines for further drug discovery. In the next year, the data we’re collecting that details our community’s experiences and preferences will directly inform the design of patient-centered clinical trials to test possible treatments that our research collaborators have identified. The first ever clinical trial for a possible treatment for CLIFAHDD Syndrome will launch soon, made possible because of the partnerships we have built with academic and government research institutions, philanthropic research foundations, and private sector partners.
JOIN US
Together, we will build on this momentum and accelerate therapeutic development for each of the NALCN-related diseases. Our research strategy is now guided by our new Director of Therapeutic Development and supported by our $2.8 Million by 2028 Raising Hope Campaign. To amplify and sustain our efforts, we invite you to join the Hope Giving Society. Reliable, recurring gifts mean we can plan boldly and fund research continuously so that we never have to pause the work our loved ones are counting on.
Across the rare disease landscape, the patient voice and the role of parent/patient advocacy groups, like the Channeling Hope Foundation, grows stronger and more influential. Three years in, we are more hopeful than ever, and more certain of the difference this community makes. Every survey response, every idea, every gift, and every hour spent in the lab brings us closer to the treatments families are waiting for. Thank you for standing with us, and for believing, alongside us, that a world free from the complications of NALCN channel-related diseases is within reach.
In Hope,
