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.
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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
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Locations
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Washington University School of Medicine
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Email: •••••@•••••