Gene therapy trial for duchenne MD halted early – what we know
NCT ID NCT05429372
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 28 times
Summary
This study tested a gene therapy called fordadistrogene movaparvovec in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to see if it was safe and could help their muscles produce a shortened version of the missing protein, dystrophin. The trial was terminated early, so results are limited. Participants had to have a confirmed genetic diagnosis and no antibodies to the virus used to deliver the therapy.
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 MUSCULAR DYSTROPHY, DUCHENNE 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
-
CTSI Clinical Research Center
Salt Lake City, Utah, 84108, United States
-
Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
-
Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
-
The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
-
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
-
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
-
The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
-
UF Health Shands Hospital
Gainesville, Florida, 32610, United States
-
University of Florida
Gainesville, Florida, 32610, United States
-
University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132, United States
-
University of Utah Hospital
Salt Lake City, Utah, 84132, United States
-
University of Utah Hospital & Clinics Investigational Drug Services
Salt Lake City, Utah, 84112, United States
-
University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108, United States
Conditions
Explore the condition pages connected to this study.