One-Time gene therapy aims to restore muscle in boys with duchenne
NCT ID NCT05693142
First seen Jun 27, 2026 · Last updated Jul 01, 2026 · Updated 2 times
Summary
This study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and walking. The trial enrolls 65 boys aged 1 to 12.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
RGX-202 (a gene therapy that delivers a microdystrophin protein to muscle cells)
What this could lead to
If successful, this could provide a one-time treatment that slows or stops muscle decline in Duchenne muscular dystrophy, helping boys maintain strength and mobility longer.
What could go wrong
This is an early-to-mid-stage trial with only 65 participants, so results may not apply to everyone. Gene therapies can cause immune reactions or other side effects, and the long-term effects are still unknown.
Disclaimer
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This is a summary of
the original study
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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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Alberta Children's Hospital
NOT_YET_RECRUITINGCalgary, Alberta, T3B 6A, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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BC Children's Hospital
RECRUITINGVancouver, British Columbia, V65 3N1, Canada
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital London Health Science Centre
RECRUITINGLondon, Ontario, Canada
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Eastern Ontario
RECRUITINGOttawa, Ontario, K1H 8L1, Canada
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Richmond at Virginia Commonwealth University
RECRUITINGRichmond, Virginia, 23298, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of the King's Daughters
RECRUITINGNorfolk, Virginia, 23510, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's
RECRUITINGCincinnati, Ohio, 45229, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oregon Health & Science University
RECRUITINGPortland, Oregon, 97239, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Stanford School of Medicine /Division of Neuromuscular Medicine
RECRUITINGPalo Alto, California, 94304, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Florida
RECRUITINGGainesville, Florida, 32610, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Massachusetts Chan Medical School
RECRUITINGWorcester, Massachusetts, 01608, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••