One-Shot hope for kids with Muscle-Wasting disease after other drugs fail
NCT ID NCT05386680
Summary
This study tested a single spinal injection of a gene therapy called OAV101 in children and teens (ages 2 to 18) with spinal muscular atrophy (SMA) who had previously stopped other SMA treatments. The main goal was to see if the one-time treatment was safe and well-tolerated. Researchers also checked if it helped improve or maintain the children's motor skills and upper body strength over one year.
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Contacts and locations
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Locations
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Boston Childrens Hospital
Boston, Massachusetts, 02215, United States
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Child Hosp Of The Kings Daughters
Norfolk, Virginia, 23507, United States
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Novartis Investigative Site
Parkville, Victoria, 3052, Australia
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Novartis Investigative Site
Leuven, 3000, Belgium
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Novartis Investigative Site
Montreal, Quebec, H4A 3J1, Canada
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Novartis Investigative Site
Bron, 69677, France
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Novartis Investigative Site
Toulouse, 31059, France
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Novartis Investigative Site
Roma, RM, 00168, Italy
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Novartis Investigative Site
Kurume, Fukuoka, 830-0011, Japan
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Novartis Investigative Site
Shinjuku Ku, Tokyo, 162 8666, Japan
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Novartis Investigative Site
Utrecht, 3584, Netherlands
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Novartis Investigative Site
Barcelona, Catalonia, 08035, Spain
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University of Wisconsin Madison Medical School
Madison, Wisconsin, 53792-7375, United States
Conditions
Explore the condition pages connected to this study.