Spinal injection gene therapy tested for rare nerve disease
NCT ID NCT05394064
Summary
This study tested a one-time gene therapy injection into the spinal fluid for adults with a rare, progressive nerve disease called adrenomyeloneuropathy (AMN). The goal was to see if delivering a working copy of a faulty gene could be safe and potentially slow down the worsening of symptoms, like walking difficulties. The trial was small and ended early, so its full effectiveness is not yet known.
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 AMN 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
-
Amsterdam UMC
Amsterdam, Netherlands
-
University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655, United States
Conditions
Explore the condition pages connected to this study.