Gene editing trial hopes to fix duchenne muscular dystrophy at its source
NCT ID NCT07429240
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tests a new gene-editing medicine called PBGENE-DMD in 18 boys aged 2 to 7 with Duchenne muscular dystrophy. The treatment aims to correct the genetic mistake that causes the disease, potentially restoring muscle strength. Researchers are first checking if it is safe and tolerable, while also looking for signs that it helps muscles produce a key protein called dystrophin.
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 WITH MUTATIONS AMENABLE TO PBGENE-DMD 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
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
-
Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63110, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••