One-Shot gene therapy tested in toddlers with devastating muscle disease
NCT ID NCT03381729
Summary
This study tested a one-time gene therapy injection for children aged 6 months to 5 years with a specific type of spinal muscular atrophy (SMA). The main goal was to find a safe dose and see if the treatment could help children gain motor skills, like standing or walking. The trial was an early safety study that enrolled 32 children who could sit up but could not stand or walk on their own.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Johns Hopkins
Baltimore, Maryland, 21287, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Nemours Children's Hospital
Orlando, Florida, 32827, United States
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Stanford University
Stanford, California, 94305, United States
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UCLA
Los Angeles, California, 90095, United States
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UT Southwestern
Dallas, Texas, 75390, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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Washington University
St Louis, Missouri, 63130, United States
Conditions
Explore the condition pages connected to this study.