One-Time gene therapy helps babies with fatal muscle disease sit independently

NCT ID NCT03461289

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

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested a one-time gene replacement therapy in 33 infants under 6 months old with spinal muscular atrophy (SMA) type 1, a severe muscle-weakening disease. The treatment aims to replace the missing SMN1 gene to improve muscle function. Key results showed that many babies could sit independently for at least 10 seconds, and most survived without needing permanent breathing support by 14 months of age.

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Conditions

The condition(s) this trial relates to.

proximal 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.

SMA

Contacts and locations

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Locations

Carlo Besta Neurological Research Institute

Milan, Italy
Great Ormond Street Hospital for Children

London, United Kingdom
Hôpital Armand Trousseau

Paris, France
Istituto Gianninia Gaslini

Genova, Italy
Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires

Liège, Belgium
Policlinico "G. Martino"

Messina, Italy
Policlinico Gemelli

Rome, Italy
The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases at Newcastle

Newcastle upon Tyne, United Kingdom
University Hospital Ghent Neuromuscular reference center

Ghent, Belgium
University of Milan

Milan, Italy

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