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Scientists search for a yardstick to measure a devastating rare disease

NCT ID NCT06018519

Summary

This study aims to prepare for future treatment trials for Creatine Transporter Deficiency (CTD), a rare genetic disorder that causes severe intellectual disability, seizures, and movement problems. Researchers will test many different questionnaires, games, and tasks on about 200 participants, including patients and healthy volunteers, to find the best ways to measure symptoms in people with significant cognitive challenges. The goal is to identify reliable tools that can detect if a future medicine is working, since standard tests often fail for these patients.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Woman, mother and child hospital, Hospices Civils de Lyon

    RECRUITING

    Bron, 69500, France

    Contact Phone: •••-•••-•••• Email: •••••@•••••

    Contact

Conditions

Explore the condition pages connected to this study.