Gene therapy hope for kids with spinal muscular atrophy after other drugs fail
NCT ID NCT05386680
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 32 times
Summary
This study tested a one-time gene therapy called OAV101 in 27 children aged 2 to 18 with spinal muscular atrophy (SMA) who had stopped taking other SMA medications. The main goals were to check safety and see if the treatment helped improve muscle strength and movement. The therapy is given as a single injection into the spinal fluid.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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
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