The SCN8A Research Grants Program is a global funding initiative advancing the discovery of safe, effective treatments for SCN8A-related disorders. Driven by families, clinicians, and researchers, we support high-quality, collaborative research that accelerates scientific progress and delivers meaningful impact for patients.
Empowering SCN8A Research
SCN8A Research Grants Program
A global collaboration dedicated to advancing the research and treatment of SCN8A-related disorders
This program is made possible through a unique international partnership among The International SCN8A Alliance, The Cute Syndrome Foundation, SCN8A España, SCN8A UK & Ireland, SCN8A Netherlands, and SCN8A France. Together, we align priorities, pool resources, and fund innovate research guided by the SCN8A Research Roadmap to ensure progress where it matters most.
SCN8A Research Roadmap
Proposed projects must align with one or more priority areas in the SCN8A Research Roadmap, outlined below. These areas represent critical opportunities to accelerate progress toward effective and safe treatments.
Transformative Therapeutics
Research focused on developing innovative therapeutic approaches for SCN8A-related disorders, including:
Novel gene therapy strategies
Safety considerations related to excessive up- or down-regulation of SCN8A expression
Effectiveness of gene therapies, including the impact of developmental timing and varying disease severity
Regulation of SCN8A expression, including transcriptional and epigenetic mechanisms
Non-Seizure Outcomes
Research aimed at understanding and addressing non-seizure manifestations of SCN8A-related disorders, including:
Defining the spectrum of non-seizure comorbidities
Improving understanding of motor, behavioral, cognitive, and other comorbidity phenotypes
Development of new animal models that better capture non-seizure outcomes
Longitudinal studies documenting progression of non-seizure comorbidities and their relationship to seizure outcomes
Improving Current Therapeutics
Research designed to optimize existing treatment approaches, including:
Improved understanding of seizure frequency and variability in SCN8A-related disorders
Identification of effective medication combinations for disease management
Application of Biomarkers
Research focused on identifying and applying biomarkers relevant to SCN8A-related disorders, including:
Identification of novel biomarkers for seizure activity and/or disease progression
Validation of biomarkers for use in research and clinical settings
Whole Brain / Whole Body Approaches
Research addressing SCN8A-related disorders at the systems level, including:
Effects of gain-of-function and loss-of-function SCN8A variants on sodium channel physiology, neuronal excitability, and synaptic transmission
Generation of human iPSC lines and isogenic control lines
Investigation of neuronal and non-neuronal cell types involved in SCN8A-related disorders
Increased understanding of circuit dynamics, synchronization, and mechanisms of epileptogenesis