New gene therapy hopes to slow duchenne muscular dystrophy in boys
NCT ID NCT05693142
First seen Nov 01, 2025 · Last updated May 15, 2026 · Updated 23 times
Summary
This study tests a one-time gene therapy called RGX-202 for boys with Duchenne muscular dystrophy (DMD). The therapy aims to help muscles work better by delivering a special protein. About 65 boys aged 1 to 12 will take part. The main goals are to check safety and see if the therapy increases dystrophin protein in muscles.
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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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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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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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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: •••••@•••••
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Children's Hospital London Health Science Centre
RECRUITINGLondon, Ontario, Canada
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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
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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
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
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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
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University of Florida
RECRUITINGGainesville, Florida, 32610, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Massachusetts Chan Medical School
RECRUITINGWorcester, Massachusetts, 01608, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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