One-Shot gene therapy aims to stop devastating muscle disease before it starts
NCT ID NCT03505099
Summary
This study tested a single, one-time gene therapy injection in infants who were diagnosed with spinal muscular atrophy (SMA) but had not yet shown symptoms. The goal was to see if delivering a working copy of a missing gene could prevent or control the severe muscle weakness and breathing problems caused by SMA. Researchers followed 30 infants to check if the treatment was safe and if it helped them reach important movement milestones like sitting and standing.
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Contacts and locations
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Locations
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Canada Childrens Hospital of Eastern Ontario
Ottawa, Ontario, K1H8L1, Canada
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Centre Hospitalier Régional Hôpital La Citadelle
Liège, 4000, Belgium
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Medical Center Dallas
Dallas, Texas, 75235, United States
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Clinic for Special Children
Strasburg, Pennsylvania, 17579, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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David Geffen School of Medicine at UCLA
Los Angeles, California, 90095, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Massachusetts General Hospital
Boston, Massachusetts, 02114, 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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St. Louis Children's Hospital
St Louis, Missouri, 63110, United States
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Sydney Children's Hospital
Randwick, New South Wales, 2145, Australia
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Tokyo Women's Medical
Tokyo, Japan
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University Hospital and UW Health Clinics
Madison, Wisconsin, 53792, United States
Conditions
Explore the condition pages connected to this study.