One-Patient trial aims to treat rare genetic brain condition
NCT ID NCT07197294
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 29 times
Summary
This study tests a personalized medicine designed for one person with a rare genetic brain disorder (NEDBA) caused by a MAPK8IP3 mutation. The treatment is an antisense oligonucleotide (ASO) that aims to improve motor skills and reduce seizures. The single participant will be followed for up to 24 months to see if the drug is safe and effective.
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 NEURODEVELOPMENTAL DISORDER WITH OR WITHOUT VARIABLE BRAIN ABNORMALITIES 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
-
Columbia University
New York, New York, 10032, United States
Conditions
Explore the condition pages connected to this study.