New gene-targeting drug aims to halt rare childhood brain disease
NCT ID NCT07215416
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 20 times
Summary
This early-stage trial tests a precision genetic medicine called atipeksen for people with Ataxia-Telangiectasia (A-T), a rare disease that damages the brain and nerves. The drug is given via spinal injection to restore a faulty gene. Up to 10 participants with a specific genetic change will be studied to see if the treatment can slow disease progression and improve daily life.
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 ATAXIA TELANGIECTASIA 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
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.