Gene-Editing breakthrough: could PBGENE-DMD fix duchenne at its source?
NCT ID NCT07429240
First seen Mar 04, 2026 · Last updated May 14, 2026 · Updated 14 times
Summary
This early-stage trial tests a one-time gene-editing treatment called PBGENE-DMD in 18 boys aged 2-7 with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and can restore dystrophin, a protein missing in DMD. The treatment aims to correct the genetic error by removing a faulty section of DNA. While not a cure, it may slow or stop disease progression.
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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.
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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