NKH Families Invited to Participate in International Rare Disease Survey
- Mar 2
- 1 min read
The NKH community has been invited to take part in an important International Rare Disease Survey focused on amplifying the voices of patients and caregivers living with rare brain signaling-related disorders — including NKH.
This global survey was developed by Hrabriša (Lil’ Brave One) in collaboration with the International Working Group of Neurotransmitter-related Disorders (iNTD). Their shared goal is simple but powerful: gather real-world patient-reported outcome measures (PROMs) to better reflect what truly matters to families.
🔗 Complete the survey here: https://www.proms-ntd.org/
Why This International Rare Disease Survey Matters
Rare disease research often centers on:
Lab values
Clinical endpoints
Measurable biomarkers
While these are important, they do not fully capture the daily lived experience of patients and caregivers.
This survey is designed to:
Identify common challenges across related rare diseases
Highlight what patients and families say actually matters most
Present these findings directly to researchers and clinicians
Strengthen patient voice in international research discussions
When families participate, the data becomes more representative — and harder to overlook.
Who Can Participate?
The questionnaire is available for:
Patients
Parents and caregivers
It has been translated into multiple languages, including:
English
Chinese
German
Greek
Spanish
French
Italian
Serbian
Turkish
Why NKH Participation Is Important
The stronger our community’s participation, the stronger our visibility within international research conversations. Collaborative efforts like this help ensure that NKH families are represented when priorities are discussed and research direction is shaped.
If you have the time and emotional bandwidth, we encourage you to complete the survey and share it with other NKH families.
Every response contributes to a clearer, stronger collective voice.
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