One-Time infusion aims to halt devastating muscle disease in toddlers
NCT ID NCT05429372
Summary
This study tested a one-time gene therapy in boys aged 2 to 4 with Duchenne muscular dystrophy (DMD). The main goals were to check if the treatment was safe and to see if it could help their muscles produce a crucial protein called dystrophin. The trial was small, with 10 participants, and has been terminated.
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Contacts and locations
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Locations
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CTSI Clinical Research Center
Salt Lake City, Utah, 84108, United States
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Perth Children's Hospital
Nedlands, Western Australia, 6009, Australia
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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The Children's Hospital at Westmead
Westmead, New South Wales, 2145, Australia
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19146, United States
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The Royal Children's Hospital Melbourne
Parkville, Victoria, 3052, Australia
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UF Health Shands Hospital
Gainesville, Florida, 32610, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital & Clinics Investigational Drug Services
Salt Lake City, Utah, 84112, United States
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University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108, United States
Conditions
Explore the condition pages connected to this study.