One-Time gene therapy helps babies with rare muscle disease sit and breathe on their own
NCT ID NCT03461289
First seen Feb 01, 2026 · Last updated May 21, 2026 · Updated 10 times
Summary
This study tested a one-time gene therapy in 33 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 SMN1 gene to help nerve cells survive. Results showed many babies could sit independently for at least 10 seconds and had better survival without needing permanent breathing support by 14 months of age.
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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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Genom att skicka in godkänner du våra Användarvillkor
Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Locations
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Carlo Besta Neurological Research Institute
Milan, Italy
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Great Ormond Street Hospital for Children
London, United Kingdom
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Hôpital Armand Trousseau
Paris, France
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Istituto Gianninia Gaslini
Genova, Italy
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Neuropédiatrie - Centre de Référence des Maladies Neuromusculaires
Liège, Belgium
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Policlinico "G. Martino"
Messina, Italy
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Policlinico Gemelli
Rome, Italy
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The John Walton Muscular Dystrophy Research Centre MRC Centre for Neuromuscular Diseases at Newcastle
Newcastle upon Tyne, United Kingdom
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University Hospital Ghent Neuromuscular reference center
Ghent, Belgium
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University of Milan
Milan, Italy
Conditions
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