A patient-driven digital infrastructure to support real-world data generation in Rett syndrome across Europe

Families. Data. Collaboration. Driving change together.
ECRD 2026
Poster #81

THE CHALLENGE

Existing real-world data on Rett syndrome is fragmented across national initiatives, research studies and clinical centres, making it difficult to obtain a comprehensive view of the patient population and their needs.

  • Fragmented datasets across national initiatives
  • Limited cross-border collaboration in research
  • Families disconnected from research opportunities
  • No European-wide view of the patient population
  • Inconsistent governance and consent processes
  • No shared infrastructure to scale future research and trials

KEY COMPONENTS

Secure Digital Infrastructure

A platform allowing caregivers to register patients and provide core demographic and diagnostic information.

Patient-Led Governance

Mechanisms led by patient organisations to ensure responsible data stewardship and transparency.

Scalable for the Future

Architecture designed to support future research, surveys and collaborations with clinicians and researchers.

Real-World Insights

Structured data ready to inform research, surveys and policy decisions, with full traceability and consent.

PATIENT-LED GOVERNANCE

  • Governed by RSE and national patient organisations
  • Transparent decision-making on data collection and access
  • Ethical use of data, aligned with the community's values
  • GDPR-compliant, privacy by design and pseudonymised at source
  • Caregivers stay in control. Consent can be reviewed or withdrawn at any time.
  • No commercial exploitation. Data access decisions are community-driven.

ROADMAP

  1. 2024 · Q4
    First pilot in Spain
  2. April 2025
    Official rettX launch
  3. 2026 · Q1
    500 patients reached in rettX
  4. 2026 · Q4
    Public dashboard & harmonised data dictionary live
  5. 2027
    Harmonised European cohort, ready for research and trials

WHAT IS rettX?

A patient-driven digital infrastructure designed to collect and manage core information about individuals living with Rett syndrome across Europe, facilitating real-world data generation while maintaining strong governance principles aligned with GDPR and patient-organisation oversight.

Families & Caregivers Patient Organisations Clinicians & Researchers Structured Real-World Data
WHAT WE COLLECT
Demographics & consent MECP2 variant Diagnostic info

EARLY IMPLEMENTATION

Pilot livewith active families & patient organisations
20+ countriescontributing patient data across Europe
Strong engagementfrom family caregivers and clinical centres
Feasibilityof a European-wide cohort demonstrated

GET INVOLVED

For families & caregivers
Register your loved one at rettx.eu. Secure, anonymous, and you stay in control of your data.
For patient organisations
Join the European network and bring your community into a shared, governed registry built for them.
For clinicians & researchers
Request access to anonymised, structured real-world data through the patient-led governance committee.

HOW DATA FLOWS THROUGH RETTX

  1. 1
    Caregivers register

    Families enter core demographic, diagnostic and functional information through a secure, GDPR-compliant interface.

  2. 2
    Structured & governed

    Data is harmonised, pseudonymised and stewarded under patient-organisation governance with full traceability and consent.

  3. 3
    Harmonised European cohort

    A single, standardised dataset emerges, ready to support cross-border research, surveys and policy work.

  4. 4
    Better outcomes

    Faster research, stronger advocacy and policy decisions grounded in real lived experience across Europe.

OUR VISION

  • One harmonised European cohort

    A single, GDPR-compliant dataset replacing 20+ fragmented national initiatives.

  • Faster, more equitable research

    Lower barriers for academic and clinical research across every European country.

  • Connected families, clinicians & researchers

    A transparent, patient-led pipeline from lived experience to research outputs.

  • A foundation for future therapies

    A research-ready cohort to support clinical trials, surveys and post-approval evidence.

FROM THE COMMUNITY

rettX is built by the community, for the community. Together, families, patient organisations, clinicians and researchers are building the foundation for a better future for everyone living with Rett syndrome.
Rett Syndrome Europe and national patient organisations
MEET US AT ECRD 2026
Poster #81 · come and discuss how rettX can support your community.
Explore the live data dashboard
rettx.eu
  • Live patient counts
  • Geographic distribution
  • Variant & age breakdowns