Scientists track rare brain disorders to unlock disease secrets
NCT ID NCT03333200
First seen Feb 10, 2026 · Last updated Apr 29, 2026 · Updated 8 times
Summary
This study follows 1500 people with rare genetic brain diseases to learn how these conditions progress over time. Researchers measure thinking, movement, and daily living skills, and look for biomarkers in blood and spinal fluid. The goal is to better understand these disorders and how treatments might affect them.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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UPMC Children's Hospital of Pittsburgh
RECRUITINGPittsburgh, Pennsylvania, 15224, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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