New study aims to unlock clues to rare brain disease in children
NCT ID NCT05659901
First seen Jan 11, 2026 · Last updated May 04, 2026 · Updated 24 times
Summary
This study is for boys aged 6 months to 17 years with Pelizaeus-Merzbacher disease (PMD), a rare genetic brain disorder. Researchers will measure changes in spinal fluid and blood markers, brain scans, and motor and thinking skills over time. The goal is to better understand how PMD progresses, which can help design future treatments. No experimental drugs are given—participants undergo tests and assessments only.
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ionis Investigative Site
RECRUITINGAtlanta, Georgia, 30342, United States
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Ionis Investigative Site
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Ionis Investigative Site
ACTIVE_NOT_RECRUITINGClermont-Ferrand, France
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Ionis Investigative Site
ACTIVE_NOT_RECRUITINGLe Kremlin-Bicêtre, 94270, France
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Ionis Investigative Site
ACTIVE_NOT_RECRUITINGGöttingen, Germany
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Ionis Investigative Site
RECRUITINGTel Aviv, 6423906, Israel
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Ionis Investigative Site
RECRUITINGMilan, 20154, Italy
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Ionis Investigative Site
RECRUITINGAmsterdam, 1081 HV, Netherlands
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Ionis Investigative Site
RECRUITINGLeeds, LS1 3EX, United Kingdom
Conditions
Explore the condition pages connected to this study.