Gene therapy trial aims to restore muscle protein in boys with duchenne
NCT ID NCT06138639
First seen Oct 31, 2025 · Last updated May 23, 2026 · Updated 25 times
Summary
This study tests a single intravenous dose of SGT-003, a gene therapy designed to help boys with Duchenne muscular dystrophy produce a shortened version of the dystrophin protein. About 60 males from infancy to under 18 years old will take part. Researchers will monitor safety and measure microdystrophin levels in muscle biopsies over five years.
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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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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611-2605, United States
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Hospital of the King's Daughters
RECRUITINGNorfolk, Virginia, 23510, United States
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITINGRome, 00168, Italy
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Great Ormond Street Hospital
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43215, United States
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Oregon Health and Sciences University
RECRUITINGPortland, Oregon, 97239, United States
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G 0A4, Canada
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University of California
RECRUITINGSan Diego, California, 92037, United States
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University of California, Davis
RECRUITINGSacramento, California, 95817, United States
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University of California, Los Angeles Medical Center
RECRUITINGLos Angeles, California, 90095, United States
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
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Conditions
Explore the condition pages connected to this study.