Gene therapy hope for toddlers with duchenne muscular dystrophy
NCT ID NCT06817382
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 30 times
Summary
This early-stage study tests a single injection of a gene therapy called INS1201 in 12 young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to see if the treatment is safe and where it goes in the body. Researchers will also look for signs that the therapy is working, such as production of a shortened version of the missing muscle protein.
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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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Rare Disease Research (USA004)
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Email: •••••@•••••
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USA001
RECRUITINGMemphis, Tennessee, 38105, United States
Contact Email: •••••@•••••
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USA002
RECRUITINGPalo Alto, California, 94070, United States
Contact Email: •••••@•••••
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USA005
RECRUITINGSan Diego, California, 93123, United States
Contact Email: •••••@•••••
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USA006
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
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USA008
RECRUITINGRochester, New York, 14642, United States
Contact Email: •••••@•••••
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USA009
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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USA010
RECRUITINGDavis, California, 95616, United States
Contact Email: •••••@•••••
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USA012
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact Email: •••••@•••••
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USA015
RECRUITINGNorfolk, Virginia, 23507, United States
Contact Email: •••••@•••••
Conditions
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