Gene therapy trial aims to slow duchenne muscular dystrophy
NCT ID NCT03368742
First seen Nov 01, 2025 · Last updated May 19, 2026 · Updated 24 times
Summary
This study tests a single dose of a gene therapy called SGT-001 in 12 boys with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and tolerable. Participants are followed for about 5 years. The therapy is given through a vein and aims to help control the disease, but it is not a cure.
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.
Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
Genom att skicka in godkänner du våra Användarvillkor
Locations
-
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095, United States
-
University of Florida
Gainesville, Florida, 32610, United States
Conditions
Explore the condition pages connected to this study.