New gene therapy hopes to fix fatal brain diseases
NCT ID NCT07445490
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy for GM2 gangliosidosis, which includes Tay-Sachs and Sandhoff diseases. Researchers will take blood cells from 6 patients, fix the genetic defect in the lab, and then see if these cells can help brain cells work better. The goal is to show the treatment is safe and effective enough to move forward.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for GM2 GANGLIOSIDOSIS are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Département de Neurologie
Paris, 75013, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New pill shows promise for rare brain disorders in early trial
- Small study tracks rare disease to pave way for future treatments
- Scientists track rare brain diseases to pave way for future cures
- New hope for rare brain diseases: long-term drug trial now recruiting
- Could a single DNA test solve the mystery of rare brain diseases in kids?
- Experimental drug zavesca tested for rare fatal brain diseases in infants