Wishes For Elliott

Education: we work to improve the understanding of scn8a by ensuring that stakeholders foster relationships break down barriers and collaborate to find treatments and improve the quality of life for all. Education is a two way street. We learn from families and we provide opportunities for families to engage with researchers pharma clinicians regulators and beyond. We break down silos that stall progress and ensure all are working with the utmost speed to reach our goal a cure for our children. We host regulr online meetings with families to educate them on the emerging science and empower them to be the most informed advocates they can be. In our work across the dees via dee-p connections, we have hosted more than 80 webinars to help families navigate caring for a loved one with major medical complexities and find support.

Advocacy : our efforts, spanning both the broad range of developmental and epileptic encephalopathies (dees) and scn8a, include diverse advocacy efforts to both legislative and administrative policymakers. Our legislative advocacy includes multiple efforts addressing pending legislative actions and appropriations that affect supported research and support for families i.e., proposed medicaid changes as well as advocating for additional federal funding for translational epilepsy research. To ensure our community is heard, we have also provided multiple comments to federal agencies, including the fda, nih, and cdc, often but not exclusively in response to published rfis. A significant efforts have included a submission of data and family stories on scn8a and a cross-dee patient listening session with the fda. Additionally, we have held a critical path innovation meeting, in collaboration with our inchstone project researchers, to ensure appropriate tools exist to measure change in those profoundly impacted by diseases in clinical trials.