Patient Listening Session with the FDA Summary
On Thursday, January 22, 2026, the CACNA1A Foundation held a Patient Listening Session with the FDA (Food and Drug Administration), the U.S. agency responsible for approving and regulating new drugs and medical interventions. This was the Foundation’s first official meeting with the FDA and marks the beginning of a partnership aimed at advancing CACNA1A-specific treatments for the community.
Patient Listening Sessions are often the first opportunity for the FDA to learn directly about a specific disease or condition. This is critical, as the agency must fully understand how a disease impacts those living with it—and what matters most to patients and their caregivers—in order to make informed decisions about new treatments. This need is especially pronounced for rare diseases like CACNA1A-related disorders.
Preparations for the CACNA1A Patient Listening Session began in the summer of 2025. Alongside Lisa Manaster, the Foundation’s Co-Founder and Board President, six caregivers were invited to share their experiences with the FDA. They were joined by Dr. Ingo Helbig from Children’s Hospital of Philadelphia, who has treated many individuals with this condition.
Through these narratives, the FDA gained insight into the profound and often devastating impact of CACNA1A-related disorders—from waking each morning to a child experiencing seizures, to severe regression following a hemiplegic migraine, to challenges with communication that can lead to distress and behavioral outbursts. Families described the risks of falls and injuries due to ataxia, as well as the widening gap in cognitive and developmental progress over time. They also highlighted the broader effects on siblings and caregivers. There was a strong emphasis on the urgent need for disease-modifying therapies, not just symptom management.
We are grateful for the opportunity to share the experiences of the CACNA1A community with the FDA. For more details, please see the Patient Listening Session summary.