Sharing Your Information
Why This Matters for the Future of NARS1 Research
We know that when you are caring for a child with a rare disorder, more paperwork is the last thing you need.
But good data is one of the biggest things standing between rare diseases and treatments. Rare-X helps researchers understand how NARS1Disorder affects people over time, which helps them design better studies and future clinical trials.
I am often asked questions like:
“Will my child talk?”
“Is this part of NARS1, or just my child?”
By sharing anonymised information in a structured way, families help clinicians and researchers build a clearer picture of NARS1. That knowledge helps families today and helps future treatments happen faster.
Natural history data helps researchers:
Understand how symptoms change over time
Compare symptoms across different variants
Know what to measure in future clinical trials
Turn real family experiences into publishable science
For example, if seizures are well documented across many families, doctors know exactly what to measure before and after testing a treatment.
Why your information matters
Where should I start?
Once you are registered, there are a lot of surveys in RARE-X and it can feel overwhelming! We also know that as a rare disease parent/caregiver, your time is valuable and precious, so we’ve created a list of the surveys to prioritize.
RARE-X Surveys to Prioritize:
If you only have time to complete one survey please do the:
Head-to-Toe Assessment (and any secondary surveys you get after completing this one)
The more information we have the better so if you can do more please complete the following additional surveys*
Vineland Adaptive Behavior Scales
ORCA (Observer-Reported Communication Ability) Measure
ABC Survey
QI-Disability Survey
CSHQ (Child Sleep Habits Questionnaire)
PediEAT (Pediatric Eating Assessment Tool)
Medication Survey
Diet Survey
CRID Alternative Participant ID Survey
Pediatric Epilepsy Learning Healthcare System (PELHS)
*These additional surveys can be found under subheadings 1) High Priority or 2) Patient Reported Data
Want more information about RARE-X or need some help getting started?
This handy video can help.
Why Rare-X?
It is a nonprofit created to accelerate rare disease research, treatments, and cures by removing barriers for data collection and sharing
RARE-X does not own, sell, or do research with the data they collect. They provide worldwide data collection that meets ethical standards
It is no cost to patients, organizations, or researchers
You own the health information you provide RARE-X
Your personal details are private
Researchers can access the de-identified health information you provide. De-identified means that researchers can see the data but they do not know who the data belongs to and it can’t be tied back to you.
It speeds up research and drug development
This can help with our current research
Allows others (researchers, drug developers, doctors) to find out more about NARS1 disease