Gene therapy breakthrough: one dose may help babies with rare muscle disease
NCT ID NCT03505099
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested a single dose of Zolgensma gene therapy in 30 infants diagnosed with spinal muscular atrophy (SMA) before symptoms appeared. The goal was to see if the treatment helps them reach motor milestones like sitting or standing alone. The therapy delivers a working copy of the missing SMN gene to stop muscle weakness from progressing.
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 (Zolgensma) gene therapy
- What this could lead to
- If successful, this one-time gene therapy could help infants with spinal muscular atrophy achieve key motor skills like sitting and standing, potentially preventing severe muscle weakness.
- What could go wrong
- This is a small, early-phase study (30 infants) and results may not apply to all SMA types. Long-term effects and risks, such as liver toxicity or immune reactions, are still being studied.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can gene therapy help babies with SMA reach milestones? a Real-World review
- School transitions put to the test for children with a rare muscle disease
- Horseback therapy may boost movement and breathing in kids with Muscle-Weakening disease
- Newborn screening study aims to catch rare diseases at birth
- Gene therapy trial aims to halt Muscle-Wasting disease
- New study explores how families cope with feeding and talking challenges in SMA type 1