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SCN8A Family Survey on Roadmap Progress

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Dear SCN8A Families,

The International SCN8A Alliance is preparing a small invitation‑only Collaborative Research Network meeting in July 2026. This one‑day meeting will bring together selected researchers, clinicians, industry scientists, and family leaders from around the world to review recent progress since the 2024 SCN8A Research Roadmap and to update research priorities for the next phase of SCN8A research.

Although the meeting itself is limited in size, your voice is essential. Your lived experience keeps research grounded in what truly matters to children, adults, and families living with SCN8A every day.

This short survey (about 10 minutes) asks for your input on the progress made so far and  what you believe priorities for future research should be.

Before taking the survey, please review this brief, plain‑language summary of progress in each of the five Roadmap area and why it matters for families. (estimated 5 minutes)

Section 1 - About Your Family

What is your name?
What is your email?
Which city, state, and country do you live in?
What is your relationship with the person living with SCN8A?
Including yourself and those you care for, what are the ages of those living with SCN8A?

Section 2 - Reactions to the Progress Summary

How well does the Roadmap Progress summary communicate the varied objectives and progress in the 5 research areas prioritized in the 2024 consensus SCN8A Research Roadmap?
How well does the Roadmap Progress summary communicate the varied objectives and progress in the 5 research areas prioritized in the 2024 consensus SCN8A Research Roadmap?
Which areas of progress feel most meaningful for your child or family?
(Select up to 3)
Are there any other important symptoms or challenges for you or your child that are missing from this summary?

Section 3 - Priorities for Future Research

Which areas would you prioritize ACROSS the 5 priority to best address the major needs and concerns for your child or family?
(1 is highest priority, 5 is lowest priority)
Transformative therapies
Non-seizure symptoms
Current treatments
Biomarkers
Whole-body understanding
Other
Which components of Transformative Therapeutics would best address the major needs and concerns for your child or family? Please rank in order of priority. (1 is highest priority)
Developing treatments that directly target SCN8A gene expression (e.g., gene editing, antisense oligonucleotides/ASOs)
Focus on SCN8A variants that are most likely to benefit from gene-targeted treatments
Moving promising treatments closer to clinical trials (improving animal models, safety testing, and dosing)
Accelerating preclinical readiness (models, dosing, safety)
Other
Which components of Non-Seizure Outcomes would best address the major needs and concerns for your child or family? Please rank in order of priority. (1 is highest priority)
GI and feeding challenges
Sensory and pain differences
Behavior and communication
Movement issues
Sleep problems
Breathing and heart-related issues
Understanding quality of life
Other
Which components of Current Therapeutics would best address the major needs and concerns for your child or family?
More evidence to guide medication selection including effective protocols for polypharmacy (use of many treatments)
Improved channels of consultation and information for rural or other clinicians with limited understanding of SCN8A
Fenfluromine, Cenobamate and other repurposed therapies
Other
Which components of Biomarkers would best address the major needs and concerns for your child or family?
Better ways to measure progress (e.g. EEG, MRI/structural, Autonomic/sleep biomarkers)
Understanding various course of disease/phenotypes
Other
Which components of Whole-Brain/Whole Body area would best address the major needs and concerns for your child or family?
SUDEP mechanisms
Multisystem involvement (GI, respiratory, behavior)
Other
Which areas would you prioritize topics WITHIN the category of Emerging, Cross-Cutting or Other topics, for example, which would best address the major needs and concerns for your child or family?
Improving integration of existing data and data sharing
Expanding cross-disciplinary collaboration/multi-disciplinary centers
Other
What symptoms or challenges have the biggest impact on your family's daily life?
What improvements would make the biggest difference for your child or family?
What topics do you most want researchers and clinicians to discuss and address at the July Collaborative Research Network meeting?
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