Gene therapy offers hope for kids with fatal brain disorder
NCT ID NCT02725580
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 28 times
Summary
This study tested a one-time gene therapy for children with a rare, severe brain disease called variant late infantile Batten disease. The treatment delivers a working copy of the CLN6 gene directly into the spinal fluid to help slow the loss of motor and language skills. Thirteen children with mild to moderate symptoms took part. The main goals were to check safety and see if the therapy could slow the disease's progression.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for VARIANT LATE-INFANTILE NEURONAL CEROID LIPOFUSCINOSIS are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Nationwide Children's Hosptial
Columbus, Ohio, 43205, United States
Conditions
Explore the condition pages connected to this study.