Gene-Editing breakthrough: could PBGENE-DMD fix duchenne at its source?
NCT ID NCT07429240
First seen Mar 04, 2026 · Last updated May 21, 2026 · Updated 15 times
Summary
This early-stage trial tests a one-time gene-editing treatment called PBGENE-DMD for boys aged 2 to 7 with Duchenne muscular dystrophy (DMD). The goal is to see if it is safe and can help muscles produce dystrophin, the protein missing in DMD. The study involves 18 participants and focuses on safety and signs of effectiveness.
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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.
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Contacts and locations
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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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