One-Time gene therapy helps babies with deadly muscle disease sit and breathe on their own

NCT ID NCT03306277

Completed Disease control Sponsor: Novartis Gene Therapies Source: ClinicalTrials.gov ↗

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.

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Conditions

The condition(s) this trial relates to.

proximal spinal muscular atrophy spinal muscular atrophy spinal muscular atrophy, type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GENE THERAPY SMA - SPINAL MUSCULAR ATROPHY

Contacts and locations

Locations

Ann and Robert H Lurie Children's Hospital

Chicago, Illinois, 60611, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
Children's Hospital Colorado

Aurora, Colorado, 80045, United States
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Columbia University

New York, New York, 10032, United States
David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States
Duke University

Durham, North Carolina, 27713, United States
Johns Hopkins Pediatric Neurology

Baltimore, Maryland, 21287, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Nemours Children's Hospital

Orlando, Florida, 32827, United States
Oregon Health and Science University

Portland, Oregon, 97239, United States
Stanford University

Stanford, California, 94305, United States
University of Texas Southwestern Medical Center

Dallas, Texas, 75235, United States
University of Utah

Salt Lake City, Utah, 84112, United States
University of Wisconsin (Madison)

Madison, Wisconsin, 53792, United States
Washington Unviersity School of Medicine

St Louis, Missouri, 63110, United States

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