Major study tracks rare brain diseases to unlock their secrets

NCT ID NCT03333200

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 1500 people with rare genetic brain disorders to learn how these diseases progress. Researchers measure thinking, movement, and daily living skills over time, and also look at brain scans and body fluids. The goal is to better understand the diseases and how treatments might help.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • UPMC Children's Hospital of Pittsburgh

    RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

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Other studies related to the condition(s) this trial covers.