Scientists map rare brain diseases to speed future cures
NCT ID NCT00668187
First seen Mar 10, 2026 · Last updated May 23, 2026 · Updated 10 times
Summary
This study follows 52 people with Tay-Sachs, Sandhoff, or GM1 gangliosidosis to learn how these rare genetic diseases progress. Researchers measure changes in development, movement, and communication skills each year. No new treatments are given; the goal is to create a clear picture of the diseases so future therapies, like gene therapy, can be tested effectively.
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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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University of Minnesota - Pediatric Genetics and Metabolism
RECRUITINGMinneapolis, Minnesota, 55455, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Conditions
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