Experimental gene therapy offers hope for children with rare fatal brain disease
NCT ID NCT07582484
First seen May 14, 2026 · Last updated May 14, 2026
Summary
This trial tests a one-time gene therapy for children with CLN6 Batten disease, a rare and fatal brain disorder. The treatment delivers a working copy of the CLN6 gene via a spinal injection. Researchers will check if it is safe and can slow the disease's progression. The study involves 12 children and includes regular check-ups over two years, plus five years of follow-up.
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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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University of California, San Diego - Rady Children's
La Jolla, California, 92093, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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