Experimental gene therapy tested in duchenne boys with immune barriers – study stopped early
NCT ID NCT06597656
First seen Nov 01, 2025 · Last updated May 08, 2026 · Updated 22 times
Summary
This early-stage study tested a gene therapy (delandistrogene moxeparvovec) in 3 boys with Duchenne muscular dystrophy who had pre-existing antibodies that could block the therapy. To overcome this, the boys first received a procedure called plasmapheresis to remove those antibodies. The main goals were to check safety and see if the therapy could produce the missing dystrophin protein. The study was terminated early, so results are limited.
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 DUCHENNE MUSCULAR DYSTROPHY 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 Hospital
Columbus, Ohio, 43205, United States
-
University of Florida, College of Medicine
Gainesville, Florida, 32610, United States
-
Washington University School of Medicine in St. Louis
St Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.