Gene therapy zolgensma tested in kids with SMA who can sit but not stand
NCT ID NCT03381729
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 1 trial tested a gene therapy called AVXS-101 (Zolgensma) in 32 children with spinal muscular atrophy (SMA) who could sit but not stand or walk. The therapy delivers a working SMN gene via a spinal injection to help improve muscle function. The study focused on safety and how well children tolerated different doses.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Onasemnogene abeparvovec-xioi (Zolgensma), a gene therapy delivered via spinal injection
What this could lead to
If successful, this could provide a treatment option for children with spinal muscular atrophy who can sit but not yet stand or walk, potentially improving motor function.
What could go wrong
This is an early phase 1 trial with only 32 participants, so results are preliminary. The trial was terminated, which may limit data. Gene therapy carries risks like immune reactions or liver issues.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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