One-Time gene therapy helps babies with deadly muscle disease sit and breathe on their own
NCT ID NCT03306277
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This phase 3 trial tested a one-time gene therapy called Zolgensma in 22 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment delivers a working copy of the missing SMN gene via an IV infusion. The main goals were to see if babies could sit independently for at least 30 seconds and survive without permanent breathing support.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
Zolgensma (onasemnogene abeparvovec-xioi), a one-time gene replacement therapy given by IV infusion
What this could lead to
If successful, this could provide a one-time treatment that helps infants with SMA type 1 sit independently and avoid the need for permanent breathing support.
What could go wrong
This is a small, single-arm trial with no comparison group, so results may not apply to all patients. Gene therapy carries risks like liver injury or immune reactions, and long-term effects are still unknown.
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 and 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 Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Columbia University
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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Duke University
Durham, North Carolina, 27713, United States
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Johns Hopkins Pediatric Neurology
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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Oregon Health and Science University
Portland, Oregon, 97239, United States
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Stanford University
Stanford, California, 94305, United States
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University of Texas Southwestern Medical Center
Dallas, Texas, 75235, United States
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University of Utah
Salt Lake City, Utah, 84112, United States
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University of Wisconsin (Madison)
Madison, Wisconsin, 53792, United States
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Washington Unviersity School of Medicine
St Louis, Missouri, 63110, United States