One-Time gene therapy aims to stop devastating muscle disease in its tracks
NCT ID NCT03505099
Summary
This study tested a single, one-time gene therapy (AVXS-101/onasemnogene abeparvovec) given to infants genetically diagnosed with spinal muscular atrophy (SMA) before they showed any symptoms. The goal was to see if this treatment could help these babies achieve major motor milestones like sitting or standing, and improve survival without needing permanent breathing support. The study followed 30 infants for up to 2 years to check the treatment's safety and effectiveness.
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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.